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    Clinical Trial Results:
    Trial of Optimal Therapy for Pseudomonas Eradication in Cystic Fibrosis

    Summary
    EudraCT number
    2009-012575-10
    Trial protocol
    GB   SE  
    Global end of trial date
    10 Apr 2018

    Results information
    Results version number
    v1(current)
    This version publication date
    10 Apr 2019
    First version publication date
    10 Apr 2019
    Other versions
    Summary report(s)
    Protocol Amendments V2.0, V4.0 and V6.0

    Trial information

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    Trial identification
    Sponsor protocol code
    CH/2007/2661
    Additional study identifiers
    ISRCTN number
    ISRCTN02734162
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Other trial identifiers
    Funder reference: HTA 07/51/01
    Sponsors
    Sponsor organisation name
    University Hospitals Bristol NHS Foundation Trust
    Sponsor organisation address
    Bristol Royal Children's Hospital, Upper Maudlin Street, Bristol, United Kingdom, BS2 8BJ
    Public contact
    Ashley Jones, Clinical Trials Research Centre, University of Liverpool, +44 151 795 8751, ctrcqa@liverpool.ac.uk
    Scientific contact
    Ashley Jones, Clinical Trials Research Centre, University of Liverpool, +44 151 795 8751, ctrcqa@liverpool.ac.uk
    Sponsor organisation name
    University of Liverpool
    Sponsor organisation address
    Research Support Office, 2nd Floor Block D, Waterhouse Building, 3 Brownlow Street, Liverpool, United Kingdom, L69 3GL
    Public contact
    Ashley Jones, Clinical Trials Research Centre, University of Liverpool, +44 151 795 8751, ctrcqa@liverpool.ac.uk
    Scientific contact
    Ashley Jones, Clinical Trials Research Centre, University of Liverpool, +44 151 795 8751, ctrcqa@liverpool.ac.uk
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    27 Sep 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    10 Apr 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    10 Apr 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    This trial assessed whether fourteen days intravenous ceftazidime with tobramycin is superior to three months (12 weeks) oral ciprofloxacin. Both treatment regimes were in conjunction with three months (12 weeks) nebulised colistin. Primary outcome: Successful eradication of P. aeruginosa infection at three months (12 weeks) after allocated treatment had started, remaining infection free through to 15 months (60 weeks) after the start of allocated treatment.
    Protection of trial subjects
    There were no formal accountability measures required for the trial. TORPEDO-CF has put the following measures in place to safeguard the safety of trial participants: - Participant completed treatment diaries to record treatment compliance - Collection of unused trial IMP - Collection of packaging of used IMP - Verbal interview conducted by the PI at the 3 month follow-up visit - Local procedures should be used if a manufacturer issues a recall
    Background therapy
    Participants recruited into the study were randomised to one of the following treatment arms: Arm A: 14 days* intravenous (IV) antibiotics as follows: - Ceftazidime 150 milligram (mg)/kilogram (kg)/day, in 3 divided doses (maximum of 3 grams (g) three times daily (tds)). Some centres used a once daily continuous infusion (where the maximum daily dose would usually be 6g/day) or twice daily regimen for ceftazidime. These centres continued to use this regimen for the study and followed their local dosing guidelines. - Tobramycin 10mg/kg/day once daily (od) (maximum 660mg /day). Some centres used a twice daily or three times daily regimen for tobramycin. These centres continued to use their current regimen for the study and should follow their local dosing guidelines. Therapeutic drug monitoring should be used to guide tobramycin dosing as per national guidelines (https://www.cysticfibrosis.org.uk/media/82010/CD_Antibiotic_treatment_for_CF_May_09.pdf) and usual clinic procedures. *Recommended treatment duration should be 14 days, minimum treatment duration should be no less than 10 days Arm B: 3 months oral ciprofloxacin twice daily (bd). (Ciprofloxacin dose will be 20 mg/kg twice daily (maximum 750mg twice daily. This is in line with the BNF for children (http://bnfc.org/bnfc/). Some clinicians prefered to use a lower dose of 15mg/kg twice daily for children under 5 years, as used in national CF guidelines (https://www.cysticfibrosis.org.uk/media/82010/CD_Antibiotic_treatment_for_CF_May_09.pdf). Both treatment arms received 3 months#of nebulised colistin in conjunction to the randomised treatment. Colistin dose was as recommended by the UK CF Trust: 1,000,000 units twice daily for children aged ≤ 2 years and 2,000,000 units twice daily for children aged > 2 years and adults. If the colistin was administered via an I-neb a lower dose of 1,000,000 units twice daily for all ages should be used.
    Evidence for comparator
    The rationale for choosing fourteen days of intravenous treatment and for choosing three months for oral treatment is that both of these are standard practice for many UK CF centres identified in the HTA feasibility study and both are standard recommendations within the published UK guideline and believed to represent current best practice.
    Actual start date of recruitment
    18 Jun 2010
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 284
    Country: Number of subjects enrolled
    Italy: 2
    Worldwide total number of subjects
    286
    EEA total number of subjects
    286
    Number of subjects enrolled per age group
    In utero
    3
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    70
    Children (2-11 years)
    161
    Adolescents (12-17 years)
    37
    Adults (18-64 years)
    15
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    60 UK based centres and 1 centre based in Italy took part in the trial. The first patient was randomised on 5th October 2010 and the last patient was randomised on 27th January 2017.

    Pre-assignment
    Screening details
    There were 1522 screenings from 1308 patients for eligibility and of those 554 were ineligible, 193 were not approached, 489 didn't provide consent and 3 patients were excluded but no reason was given. 286 patients were randomised into the trial. (Note that patients could be screened more than once)

    Period 1
    Period 1 title
    Baseline
    Is this the baseline period?
    Yes
    Allocation method
    Randomised - controlled
    Blinding used
    Not blinded
    Blinding implementation details
    Randomisation lists were generated in a 1:1 ratio using simple block randomisation with random variable block lengths. Factors within the protocol that were used to stratify randomisation were not disclosed to prevent prediction in this open trial.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Intravenous (IV) antibiotics
    Arm description
    14 days IV antibiotics; Ceftazidime and Tobramycin alongside three months of nebulised Colistin
    Arm type
    Experimental

    Investigational medicinal product name
    Ceftazidime
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Ceftazidime 150 milligram (mg)/kilogram (kg)/day, in 3 divided doses (maximum of 3 grams (g) three times daily (tds)). Some centres used a once daily continuous infusion (where the maximum daily dose would usually be 6g/day) or twice daily regimen for ceftazidime.

    Investigational medicinal product name
    Tobramycin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Tobramycin 10mg/kg/day once daily (od) (maximum 660mg /day). Some centres used a twice daily or three times daily regimen for tobramycin.

    Investigational medicinal product name
    Colistin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nebuliser solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    3 months (12 weeks) of nebulised colistin in conjunction to the randomised treatment. Colistin dose was as recommended by the UK CF Trust: 1,000,000 units twice daily for children aged ≤ 2 years and 2,000,000 units twice daily for children aged > 2 years and adults. If the colistin is administered via an I-neb a lower dose of 1,000,000 units twice daily for all ages was used.

    Arm title
    Oral antibiotic therapy
    Arm description
    3 months (12 weeks) of oral ciprofloxacin alongside 3 months (12 weeks) of nebulised colistin.
    Arm type
    Active comparator

    Investigational medicinal product name
    Ciprofloxacin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Granules and solvent for oral suspension, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    3 months (12 weeks) oral ciprofloxacin twice daily (bd). (Ciprofloxacin dose was 20 mg/kg twice daily (maximum 750mg twice daily. Some clinicians could use a lower dose of 15mg/kg twice daily for children under 5 years, as used in national CF guidelines.

    Investigational medicinal product name
    Colistin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nebuliser solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    3 months (12 weeks) of nebulised colistin in conjunction to the randomised treatment. Colistin dose was as recommended by the UK CF Trust: 1,000,000 units twice daily for children aged ≤ 2 years and 2,000,000 units twice daily for children aged > 2 years and adults. If the colistin is administered via an I-neb a lower dose of 1,000,000 units twice daily for all ages was used.

    Number of subjects in period 1
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Started
    137
    149
    Completed
    137
    148
    Not completed
    0
    1
         Unable to obtain PI sign-off
    -
    1
    Period 2
    Period 2 title
    Follow-up
    Is this the baseline period?
    No
    Allocation method
    Not applicable
    Blinding used
    Not blinded
    Blinding implementation details
    See blinding details for the baseline period.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Intravenous (IV) antibiotics
    Arm description
    14 days IV antibiotics; Ceftazidime and Tobramycin alongside three months nebulised Colistin
    Arm type
    Experimental

    Investigational medicinal product name
    Ceftazidime
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Ceftazidime 150 milligram (mg)/kilogram (kg)/day, in 3 divided doses (maximum of 3 grams (g) three times daily (tds)). Some centres used a once daily continuous infusion (where the maximum daily dose would usually be 6g/day) or twice daily regimen for ceftazidime.

    Investigational medicinal product name
    Tobramycin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Tobramycin 10mg/kg/day once daily (od) (maximum 660mg /day). Some centres used a twice daily or three times daily regimen for tobramycin.

    Investigational medicinal product name
    Colistin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nebuliser solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    3 months (12 weeks) of nebulised colistin in conjunction to the randomised treatment. Colistin dose was as recommended by the UK CF Trust: 1,000,000 units twice daily for children aged ≤ 2 years and 2,000,000 units twice daily for children aged > 2 years and adults. If the colistin is administered via an I-neb a lower dose of 1,000,000 units twice daily for all ages was used.

    Arm title
    Oral antibiotic therapy
    Arm description
    3 months (12 weeks) of oral ciprofloxacin alongside 3 months (12 weeks) of nebulised colistin.
    Arm type
    Active comparator

    Investigational medicinal product name
    Ciprofloxacin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    3 months (12 weeks) oral ciprofloxacin twice daily (bd). (Ciprofloxacin dose was 20 mg/kg twice daily (maximum 750mg twice daily. Some clinicians preferred to use a lower dose of 15mg/kg twice daily for children under 5 years, as used in national CF guidelines.

    Investigational medicinal product name
    Colistin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Nebuliser solution
    Routes of administration
    Inhalation use
    Dosage and administration details
    3 months (12 weeks) of nebulised colistin in conjunction to the randomised treatment. Colistin dose was as recommended by the UK CF Trust: 1,000,000 units twice daily for children aged ≤ 2 years and 2,000,000 units twice daily for children aged > 2 years and adults. If the colistin is administered via an I-neb a lower dose of 1,000,000 units twice daily for all ages was used.

    Number of subjects in period 2
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Started
    137
    148
    Completed
    132
    132
    Not completed
    5
    16
         Other
    1
    2
         Adverse event, non-fatal
    -
    1
         Consent withdrawn by subject
    1
    4
         Lost to follow-up
    3
    9

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Intravenous (IV) antibiotics
    Reporting group description
    14 days IV antibiotics; Ceftazidime and Tobramycin alongside three months of nebulised Colistin

    Reporting group title
    Oral antibiotic therapy
    Reporting group description
    3 months (12 weeks) of oral ciprofloxacin alongside 3 months (12 weeks) of nebulised colistin.

    Reporting group values
    Intravenous (IV) antibiotics Oral antibiotic therapy Total
    Number of subjects
    137 149 286
    Age categorical
    PLEASE NOTE THAT THE AGE FOR 3 PARTICIPANTS COULD NOT BE CALCULATED AND THEREFORE DATA WAS UNOBTAINABLE FOR THESE PARTICIPANTS. THE TABLE TITLED 'AGE GROUP BREAKDOWN FOR TRIAL' IN THE SECTION 'TRIAL INFORMATION' HAS THESE 3 PARTICIPANTS ADDED IN THE 'IN UTERO' SECTION TO ALLOW RESULTS TO BE POSTED ONTO EUDRACT. AGE DATA ARE ONLY AVAILABLE FOR 283 PARTICIPANTS IN THE TRIAL.
    Units: Subjects
        In utero
    0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0
        Newborns (0-27 days)
    0 0 0
        Infants and toddlers (28 days-23 months)
    42 28 70
        Children (2-11 years)
    71 90 161
        Adolescents (12-17 years)
    18 19 37
        Adults (18-64 years)
    6 9 15
        From 65-84 years
    0 0 0
        85 years and over
    0 0 0
        Missing
    0 2 2
        Not recorded
    0 1 1
    Gender categorical
    Units: Subjects
        Female
    74 81 155
        Male
    63 67 130
        Not recorded
    0 1 1
    Pseudomonas status
    Units: Subjects
        Free
    56 55 111
        Naïve
    81 93 174
        Not recorded
    0 1 1
    Genotype
    Units: Subjects
        Delta F508/ Delta F508
    70 90 160
        Delta F508/other
    40 43 83
        Delta F508/unknown
    4 5 9
        other/other
    12 7 19
        Diagnosis not based on genetics
    10 3 13
        Unknown whether diagnosis based on genetics
    1 0 1
        Not recorded
    0 1 1
    Pulmonary exacerbation
    Units: Subjects
        Yes
    18 17 35
        No
    119 131 250
        Not recorded
    0 1 1
    Candida detected
    Units: Subjects
        Yes
    11 17 28
        No
    126 131 257
        Not recorded
    0 1 1
    MRSA detected
    Units: Subjects
        Yes
    0 2 2
        No
    137 146 283
        Not recorded
    0 1 1
    Burkholderia cepacia detected
    Units: Subjects
        Yes
    0 0 0
        No
    137 148 285
        Not recorded
    0 1 1
    Aspergillus detected
    Units: Subjects
        Yes
    2 2 4
        No
    135 146 281
        Not recorded
    0 1 1
    Other micro-organism detected
    Units: Subjects
        Yes
    26 31 57
        No
    111 117 228
        Not recorded
    0 1 1
    BMI z-score (paediatric)
    Units: z-score
        arithmetic mean (standard deviation)
    0.3 ± 1 0.3 ± 0.9 -
    BMI (adults)
    Units: (m/kg^2)
        arithmetic mean (standard deviation)
    24.6 ± 1.8 23.2 ± 2.3 -
    Time from PsA isolation to treatment initiation
    Units: (days)
        arithmetic mean (standard deviation)
    9 ± 5 7 ± 5 -
    %predicted FEV1
    Units: FEV1 %predicted
        arithmetic mean (standard deviation)
    86.6 ± 15.8 85.7 ± 16 -
    %predicted FVC
    Units: FVC %predicted
        arithmetic mean (standard deviation)
    92.2 ± 15.5 95.1 ± 14.5 -
    %predicted FEF25-75
    Units: FEF25-75 %predicted
        arithmetic mean (standard deviation)
    72.7 ± 26.6 70.6 ± 30.3 -
    Oxygen saturation
    Units: (%)
        arithmetic mean (standard deviation)
    97.7 ± 1.4 97.7 ± 1.7 -

    End points

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    End points reporting groups
    Reporting group title
    Intravenous (IV) antibiotics
    Reporting group description
    14 days IV antibiotics; Ceftazidime and Tobramycin alongside three months of nebulised Colistin

    Reporting group title
    Oral antibiotic therapy
    Reporting group description
    3 months (12 weeks) of oral ciprofloxacin alongside 3 months (12 weeks) of nebulised colistin.
    Reporting group title
    Intravenous (IV) antibiotics
    Reporting group description
    14 days IV antibiotics; Ceftazidime and Tobramycin alongside three months nebulised Colistin

    Reporting group title
    Oral antibiotic therapy
    Reporting group description
    3 months (12 weeks) of oral ciprofloxacin alongside 3 months (12 weeks) of nebulised colistin.

    Primary: Primary efficacy assessment - Successful eradication of P. aeruginosa three months after the start of treatment, remaining infection free through to 15 months after the start of treatment

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    End point title
    Primary efficacy assessment - Successful eradication of P. aeruginosa three months after the start of treatment, remaining infection free through to 15 months after the start of treatment
    End point description
    End point type
    Primary
    End point timeframe
    3 months after the start of treatment through to 15 months after the start of treatment .
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    125
    130
    Units: Subjects
        Successful eradication
    55
    68
        Unsuccessful eradication
    70
    62
    Statistical analysis title
    Successful eradication from 3 months to 15 months
    Statistical analysis description
    The number of patients who were classified as (a) a success and (b) failure for the primary outcome (and percentages) were presented for each treatment arm. The relative risk together with 95% confidence interval was reported along with a two-sided p-value from a chi-squared test.
    Comparison groups
    Oral antibiotic therapy v Intravenous (IV) antibiotics
    Number of subjects included in analysis
    255
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.184
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    0.84
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.65
         upper limit
    1.09

    Primary: Primary efficacy assessment - Successful eradication of P. aeruginosa three months after the start of treatment, remaining infection free through to 15 months after the start of treatment

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    End point title
    Primary efficacy assessment - Successful eradication of P. aeruginosa three months after the start of treatment, remaining infection free through to 15 months after the start of treatment
    End point description
    End point type
    Primary
    End point timeframe
    From 3 months after the start of treatment to 15 months after the start of treatment.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    110
    116
    Units: Subjects
        Successful eradication
    97
    111
        Unsuccessful eradication
    13
    5
    Statistical analysis title
    Unsuccessful eradication at 3 months
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    226
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.037
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    2.74
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.01
         upper limit
    7.44

    Primary: Primary efficacy assessment – Sensitivity analysis 6: no T15 window (Post hoc)

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    End point title
    Primary efficacy assessment – Sensitivity analysis 6: no T15 window (Post hoc)
    End point description
    End point type
    Primary
    End point timeframe
    From 3 months after the start of treatment to 15 months after the start of treatment.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    136
    144
    Units: Subjects
        Successful eradication
    58
    70
        Unsuccessful eradication
    78
    74
    Statistical analysis title
    Sensitivity analysis 6
    Statistical analysis description
    The relative risk with 95% CI was presented. A chi-squared test will be used to calculate a p-value for this relative risk.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    280
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.317
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    0.88
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.68
         upper limit
    1.13

    Secondary: Time to reoccurrence of original P.aeruginosa infection - unknown strains assumed to be same as baseline

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    End point title
    Time to reoccurrence of original P.aeruginosa infection - unknown strains assumed to be same as baseline
    End point description
    End point type
    Secondary
    End point timeframe
    3 months to 24 months. Only patients who have a baseline sample and at least one sample post three months will be included in this analysis.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    137
    148
    Units: Subjects
        Reoccurence of original P.aeruginosa infection
    74
    66
        Censored
    63
    82
    Statistical analysis title
    Time to reoccurrence of original P.aeruginosa
    Statistical analysis description
    Difference between the two treatment arms was tested using the log-rank test. Cox proportional hazards regression was used to calculate a hazard ratio with 95% CI, comparing the IV treatment group to oral, – unknown strains assumed to be same as baseline.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    285
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.061
    Method
    Logrank
    Parameter type
    Hazard ratio (HR)
    Point estimate
    1.37
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.99
         upper limit
    1.91

    Secondary: Time to reoccurrence of original P.aeruginosa infection - unknown strains assumed to be different to baseline

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    End point title
    Time to reoccurrence of original P.aeruginosa infection - unknown strains assumed to be different to baseline
    End point description
    End point type
    Secondary
    End point timeframe
    From 3 to 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    137
    148
    Units: Subjects
        Reoccurence of original P.aeruginosa infection
    21
    14
        Censored
    116
    134
    Statistical analysis title
    Time to reoccurrence of original P.aeruginosa
    Statistical analysis description
    Difference between the two treatment arms was tested using the log-rank test. Cox proportional hazards regression was used to calculate a hazard ratio with 95% CI, comparing the IV treatment group to oral, – unknown strains assumed to be differnt than baseline.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    285
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.075
    Method
    Logrank
    Parameter type
    Hazard ratio (HR)
    Point estimate
    1.85
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.94
         upper limit
    3.64

    Secondary: Re-infection with a different strain of P.aeruginosa

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    End point title
    Re-infection with a different strain of P.aeruginosa
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 15 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    25
    17
    Units: Subjects
        Same genotype
    19
    12
        Different genotype
    6
    5
    Statistical analysis title
    Re-infection with a different strain
    Statistical analysis description
    The relative risk together with 95% confidence interval was reported along with a two-sided p-value from a chi-squared test.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    42
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.733
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    0.82
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.3
         upper limit
    2.25

    Secondary: Post hoc analysis – Genotyping results

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    End point title
    Post hoc analysis – Genotyping results
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    28
    22
    Units: Subjects
        Same genotype
    20
    13
        Different genotype
    8
    9
    No statistical analyses for this end point

    Secondary: Lung function - FEV1% predicted

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    End point title
    Lung function - FEV1% predicted
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    67 [1]
    70 [2]
    Units: FEV1% predicted
    arithmetic mean (standard deviation)
        Baseline
    86.6 ± 15.8
    85.7 ± 16.0
        3 Months (11-13 weeks)
    86.3 ± 18.6
    81.9 ± 15.6
        15 Months (59-62 weeks)
    89.5 ± 18.2
    82.3 ± 18.9
        24 Months (95-97 weeks)
    83.8 ± 13.7
    79.8 ± 15.0
    Notes
    [1] - At baseline n=67, T3 n=25, T15 n=32, T24 n= 11
    [2] - At baseline n=70, T3 n=28, T15 n=32 , T24 n=17
    Statistical analysis title
    FEV1 % predicted - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    137
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.008
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    3.75
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.97
         upper limit
    6.53
    Statistical analysis title
    FEV1 % predicted - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    137
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.184
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    2.08
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.99
         upper limit
    5.14
    Statistical analysis title
    FEV1 % predicted - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures will be fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    137
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.705
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    0.82
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.45
         upper limit
    5.1
    Statistical analysis title
    FEV1 % predicted - treat difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a continuous variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    137
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.019
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    3.16
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.51
         upper limit
    5.8

    Secondary: Lung function - FVC% predicted

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    End point title
    Lung function - FVC% predicted
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    67 [3]
    70 [4]
    Units: FVC% predicted
    arithmetic mean (standard deviation)
        Baseline
    92.2 ± 15.5
    95.1 ± 14.5
        3 Months (11-13 weeks)
    89.7 ± 19.3
    88.9 ± 16.2
        15 months (59-62 weeks)
    91.4 ± 18.4
    89.4 ± 18.8
        24 Months (95-97 weeks)
    89.4 ± 15.4
    91.8 ± 20.4
    Notes
    [3] - At baseline n=67, T3 n= 25, T15 n=32 and T24 n=11
    [4] - At baseline n=70, T3 n=28, T15 n=32 and T24 n=17
    Statistical analysis title
    FVC % predicted - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    137
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.008
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    3.86
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    1.03
         upper limit
    6.69
    Statistical analysis title
    FVC % predicted - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    137
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.0396
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    3.14
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.15
         upper limit
    6.14
    Statistical analysis title
    FVC % predicted - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    137
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.216
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    2.61
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.52
         upper limit
    6.73
    Statistical analysis title
    FVC % predicted - treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a continuous variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    137
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.009
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    3.56
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.9
         upper limit
    6.21

    Secondary: Lung function - FEF25-75% predicted

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    End point title
    Lung function - FEF25-75% predicted
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    44 [5]
    53 [6]
    Units: FEF25-75% predicted
    arithmetic mean (standard deviation)
        Baseline
    72.7 ± 26.6
    70.6 ± 30.3
        3 Months (11-13 weeks)
    84.0 ± 23.5
    70.6 ± 25.1
        15 Months (59-62 weeks)
    77.2 ± 28.0
    68.1 ± 21.5
        24 Months (95-97 weeks)
    58.3 ± 23.1
    58.9 ± 13.3
    Notes
    [5] - At baseline n=44, T3 n=18, T15 n=26 and T24 n=7
    [6] - At baseline n=53, T3 n=20, T15 n=21 and T24 n=12
    Statistical analysis title
    FEV25-75% predicted - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    97
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.274
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    3.76
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.99
         upper limit
    10.52
    Statistical analysis title
    FEV25-75% predicted - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    97
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.345
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    3.46
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.74
         upper limit
    10.66
    Statistical analysis title
    FEV25-75% predicted - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    97
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.505
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    3.23
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.29
         upper limit
    12.76
    Statistical analysis title
    FEV25-75% predicted - treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a continuous variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    97
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.269
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    3.64
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.82
         upper limit
    10.11

    Secondary: Oxygen saturation

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    End point title
    Oxygen saturation
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    118 [7]
    133 [8]
    Units: Percentage
    arithmetic mean (standard deviation)
        baseline
    97.7 ± 1.4
    97.7 ± 1.7
        3 months (11-13 weeks)
    97.6 ± 1.5
    98.2 ± 1.6
        15 months (59-62 weeks)
    97.8 ± 1.5
    97.9 ± 1.4
        24 months (95-97 weeks)
    97.9 ± 1.1
    97.6 ± 1.8
    Notes
    [7] - At baseline n=118, T3 n= 42 , t15 n=48 and T24 n=17
    [8] - At baseline n=133, T3 n=53, T15 n=46 and T24 n=26
    Statistical analysis title
    O2 Saturation - 3 month treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    251
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.728
    Method
    Mixed models analysis
    Parameter type
    3 Month Treatment difference
    Point estimate
    0.05
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.21
         upper limit
    0.3
    Statistical analysis title
    O2 Saturation - 15 month treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    251
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.709
    Method
    Mixed models analysis
    Parameter type
    15 Month Treatment difference
    Point estimate
    0.05
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.2
         upper limit
    0.29
    Statistical analysis title
    O2 Saturation - Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a continuous variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    251
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.686
    Method
    Mixed models analysis
    Parameter type
    Treatment difference
    Point estimate
    0.05
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.18
         upper limit
    0.27
    Statistical analysis title
    O2 Saturation - 24 Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    251
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.79
    Method
    Mixed models analysis
    Parameter type
    24 month Treatment difference
    Point estimate
    0.05
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.3
         upper limit
    0.4

    Secondary: Growth and nutritional status - Height z-scores

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    End point title
    Growth and nutritional status - Height z-scores
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    125 [9]
    131 [10]
    Units: Height z-scores
    arithmetic mean (standard deviation)
        Baseline
    -0.4 ± 1.0
    -0.4 ± 1.0
        3 Months (11-13 weeks)
    -0.2 ± 1.0
    -0.2 ± 1.0
        15 Months (59-62 weeks)
    -0.2 ± 1.0
    -0.1 ± 0.9
        24 Months (95-97 weeks)
    -0.5 ± 0.8
    -0.7 ± 0.6
    Notes
    [9] - At baseline n=125, T3 n=32, T15 n=36 and T24 n=13
    [10] - At baseline n=131, T3 n=47, T15 n=32 and T24 n=16
    Statistical analysis title
    Height z-scores - 3 Month Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures will be fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    256
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.952
    Method
    Mixed models analysis
    Parameter type
    3 Month treatment difference
    Point estimate
    0
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.06
         upper limit
    0.05
    Statistical analysis title
    Height z-scores - 15 Month Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    256
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.572
    Method
    Mixed models analysis
    Parameter type
    15 Month treatment difference
    Point estimate
    -0.03
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.13
         upper limit
    0.07
    Statistical analysis title
    Height z-scores - 24 Month Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    256
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.543
    Method
    Mixed models analysis
    Parameter type
    24 Month treatment difference
    Point estimate
    -0.05
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.21
         upper limit
    0.11
    Statistical analysis title
    Height z-scores - Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a continuous variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    256
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.939
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    0
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.06
         upper limit
    0.05

    Secondary: Growth and nutritional status - Weight z-scores

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    End point title
    Growth and nutritional status - Weight z-scores
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    102 [11]
    109 [12]
    Units: Weight z-scores
    arithmetic mean (standard deviation)
        Baseline
    0.0 ± 1.1
    0.0 ± 0.9
        3 Months (11-13 weeks)
    0.2 ± 1.2
    0.2 ± 0.9
        15 Months (59-62 weeks)
    0.1 ± 1.1
    0.3 ± 0.8
        24 Months (95-97 weeks)
    0.1 ± 0.7
    -0.4 ± 0.8
    Notes
    [11] - At baseline n=102, T3 n=30, T15 n=27, T24 n=12
    [12] - At baseline n=109, T3 n=37, T15 n=23, T24 n=11
    Statistical analysis title
    Weight z-scores - 3 Month Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    211
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.988
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    0
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.06
         upper limit
    0.06
    Statistical analysis title
    Weight z-scores - 15 Month Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    211
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.794
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    -0.02
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.15
         upper limit
    0.11
    Statistical analysis title
    Weight z-scores - 24 Month Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    211
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.78
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    -0.03
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.24
         upper limit
    0.18
    Statistical analysis title
    Weight z-scores - Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a continuous variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    211
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.987
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    0
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.06
         upper limit
    0.06

    Secondary: Growth and nutritional status - BMI z-scores

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    End point title
    Growth and nutritional status - BMI z-scores
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    125 [13]
    131 [14]
    Units: BMI z-scores
    arithmetic mean (standard deviation)
        Baseline
    0.3 ± 1.0
    0.3 ± 0.9
        3 Months (11-13 weeks)
    0.3 ± 1.0
    0.2 ± 1.0
        15 Months (59-62 weeks)
    0.4 ± 1.1
    0.2 ± 1.1
        24 Months (95-97 weeks)
    0.5 ± 0.7
    0.0 ± 0.8
    Notes
    [13] - At baseline n=125, T3 n=32, T15 n=36 and T24 n=13
    [14] - At baseline n=131, T3 n=47, T15 n=32 and T24 n=16
    Statistical analysis title
    BMI z-score - 3 Month Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    256
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.767
    Method
    Mixed models analysis
    Parameter type
    3 Month Treatment Difference
    Point estimate
    -0.01
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.08
         upper limit
    0.06
    Statistical analysis title
    BMI z-score - 15 Month Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    256
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.912
    Method
    Mixed models analysis
    Parameter type
    15 Month Treatment Difference
    Point estimate
    0.01
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.14
         upper limit
    0.16
    Statistical analysis title
    BMI z-score - 24 Month Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    256
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.854
    Method
    Mixed models analysis
    Parameter type
    24 Month Treatment Difference
    Point estimate
    0.02
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.22
         upper limit
    0.26
    Statistical analysis title
    BMI z-score - Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a continuous variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    256
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.774
    Method
    Mixed models analysis
    Parameter type
    Treatment Difference
    Point estimate
    -0.01
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.08
         upper limit
    0.06

    Secondary: Growth and nutritional status - BMI

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    End point title
    Growth and nutritional status - BMI
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    7 [15]
    10 [16]
    Units: m/kg^2
    arithmetic mean (standard deviation)
        Baseline
    24.2 ± 1.9
    22.9 ± 2.4
        3 Months (11-13 weeks)
    23.1 ± 2.7
    21.0 ± 0.6
        15 Months (59-62 weeks)
    26.4 ± 1.4
    22.2 ± 1.4
        24 Months (95-97 weeks)
    25.2 ± 1.1
    22.8 ± 2.8
    Notes
    [15] - At baseline n=7, T3 n=2, T15 n=2 and T24 n=2
    [16] - At baseline n=10, T3 n=2, T15 n=3 and T24 n=3
    Statistical analysis title
    BMI - 3 Month Treat Diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    17
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.067
    Method
    Mixed models analysis
    Parameter type
    3 Month Treatment Difference
    Point estimate
    -0.48
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1
         upper limit
    0.03
    Statistical analysis title
    BMI - 15 Month Treat Diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    17
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.029
    Method
    Mixed models analysis
    Parameter type
    15 Month Treatment Difference
    Point estimate
    -0.73
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.39
         upper limit
    -0.08
    Statistical analysis title
    BMI - Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a continuous variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    17
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.02
    Method
    Mixed models analysis
    Parameter type
    Treatment Difference
    Point estimate
    -0.56
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.03
         upper limit
    -0.09
    Statistical analysis title
    BMI - 24 Month Treat Diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a continuous variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    17
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.093
    Method
    Mixed models analysis
    Parameter type
    24 Month Treatment Difference
    Point estimate
    -0.92
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.01
         upper limit
    0.16

    Secondary: Number of pulmonary exacerbations - median number of exacerbations during the 15 months following treatment commencement

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    End point title
    Number of pulmonary exacerbations - median number of exacerbations during the 15 months following treatment commencement
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 15 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    137
    146
    Units: Number of pulmonary exacerbations
        median (inter-quartile range (Q1-Q3))
    0 (0 to 1)
    0 (0 to 1)
    Statistical analysis title
    Mann–Whitney U test
    Statistical analysis description
    A Mann-Whitney U-test will test whether the distribution of number of exacerbations is the same in each treatment arm
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    283
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.09
    Method
    Wilcoxon (Mann-Whitney)
    Confidence interval

    Secondary: Number of pulmonary exacerbations - Number of participants experiencing at least one exacerbation during the first 15 months of follow up

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    End point title
    Number of pulmonary exacerbations - Number of participants experiencing at least one exacerbation during the first 15 months of follow up
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 15 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    137
    146
    Units: Subjects
        0 exacerbations
    99
    94
        >=1 exacerbations
    38
    52
    Statistical analysis title
    Chi-squared test
    Statistical analysis description
    The number and percentage of patients experiencing at least one exacerbation in each treatment arm was reported. Treatment groups were compared using the chi-squared test.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    283
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.155
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    0.78
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.55
         upper limit
    1.1

    Secondary: Admission to hospital - Number of participants experiencing at least one hospital stay during the first 3 months of treatment

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    End point title
    Admission to hospital - Number of participants experiencing at least one hospital stay during the first 3 months of treatment
    End point description
    End point type
    Secondary
    End point timeframe
    First 3 months of treatment.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    135
    143
    Units: Subjects
        0 Hospital Stays
    110
    128
        >=1 Hospital Stay
    25
    15
    Statistical analysis title
    Admission to hospital - 3 months of treatment
    Statistical analysis description
    Number of participants experiencing at least one hospital stay during the first 3 months of treatment
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    278
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.057
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    1.77
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.97
         upper limit
    3.2

    Secondary: Admission to hospital - Number of participants experiencing at least one hospital stay during the 12 months following treatment

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    End point title
    Admission to hospital - Number of participants experiencing at least one hospital stay during the 12 months following treatment
    End point description
    End point type
    Secondary
    End point timeframe
    12 months following treatment.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    129
    136
    Units: Subjects
        0 Hospital Stays
    89
    75
        >=1 Hospital Stay
    40
    61
    Statistical analysis title
    Admission to hospital - 12 months
    Statistical analysis description
    Number of participants experiencing at least one hospital stay during the 12 months following treatment.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    265
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.02
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    0.69
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.5
         upper limit
    0.95

    Secondary: Admission to hospital - Number of participants experiencing at least one hospital stay between 15 and 24 months

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    End point title
    Admission to hospital - Number of participants experiencing at least one hospital stay between 15 and 24 months
    End point description
    Number of participants experiencing at least one hospital stay between 15 months and 24 months.
    End point type
    Secondary
    End point timeframe
    Between 15 months and 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    105
    107
    Units: Subjects
        0 Hospital Stays
    72
    66
        >=1 Hospital Stay
    33
    41
    Statistical analysis title
    Admission to hospital - 15-24 months
    Statistical analysis description
    Number of participants experiencing at least one hospital stay between 15 months and 24 months
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    212
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.293
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    0.82
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.57
         upper limit
    1.19

    Secondary: Number of days spent as an inpatient in hospital - During treatment phase

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    End point title
    Number of days spent as an inpatient in hospital - During treatment phase
    End point description
    End point type
    Secondary
    End point timeframe
    From baseline to 3 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    135
    143
    Units: Days
        median (inter-quartile range (Q1-Q3))
    0 (0 to 0)
    0 (0 to 0)
    Statistical analysis title
    Length of stay in days during the treatment phase
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    278
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.066
    Method
    Wilcoxon (Mann-Whitney)
    Confidence interval

    Secondary: Number of days spent as an inpatient in hospital - During the 12 months post treatment phase

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    End point title
    Number of days spent as an inpatient in hospital - During the 12 months post treatment phase
    End point description
    End point type
    Secondary
    End point timeframe
    12 months post treatment.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    129
    136
    Units: days
        median (inter-quartile range (Q1-Q3))
    0 (0 to 3)
    0 (0 to 13)
    Statistical analysis title
    Length of stay during 12 months post treatment
    Statistical analysis description
    Length of stay in days during the 12 months post treatment phase.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    265
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.005
    Method
    Wilcoxon (Mann-Whitney)
    Confidence interval

    Secondary: Number of days spent as an inpatient in hospital - between 15 and 24 months follow up

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    End point title
    Number of days spent as an inpatient in hospital - between 15 and 24 months follow up
    End point description
    End point type
    Secondary
    End point timeframe
    Between 15 and 24 months.
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    99
    102
    Units: days
        median (inter-quartile range (Q1-Q3))
    0 (0 to 2.8)
    0 (0 to 4)
    Statistical analysis title
    Length of stay 15-24 months
    Statistical analysis description
    Length of stay in days, between 15 and 24 months follow up.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    201
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.261
    Method
    Wilcoxon (Mann-Whitney)
    Confidence interval

    Secondary: CFQ - Physical functioning – Self Report

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    End point title
    CFQ - Physical functioning – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    Basline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    53 [17]
    61 [18]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    80.70 ± 19.8
    81 ± 17.4
        3 months (11-13 weeks)
    85.5 ± 15.1
    78.1 ± 20
        15 months (59-62 weeks)
    83 ± 18
    85.5 ± 14.7
        24 months (95-97 weeks)
    84.2 ± 14.6
    86.2 ± 16.7
    Notes
    [17] - The number of patients at base line was 53, 49 at 3 months, 50 at 15 months and 44 at 24 months.
    [18] - The number of patients at baseline was 61, 56 at 3 months, 50 at 15 months and 45 at 24 months
    Statistical analysis title
    Physical functioning (Self-report) - 3 Month Diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups at T3 (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.056
    Method
    Mixed models analysis
    Parameter type
    Mean difference at 3 months
    Point estimate
    6.63
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.18
         upper limit
    13.44
    Statistical analysis title
    Physical functioning (Self-report) - 15 month diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups at T15 (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.292
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    -3.63
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -10.41
         upper limit
    3.16
    Statistical analysis title
    Physical functioning (Self-report) - 24 Month diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups at T24 (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.793
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    0.94
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.15
         upper limit
    8.03
    Statistical analysis title
    Physical functioning (Self-report) - Treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable . The following was reported: mean (SD) for each treatment group; mean (95% CI) and difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.538
    Method
    Mixed models analysis
    Parameter type
    Treatment Difference
    Point estimate
    1.62
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.57
         upper limit
    6.8

    Secondary: CFQ - Role/school functioning – Self Report

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    End point title
    CFQ - Role/school functioning – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    14 [19]
    18 [20]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    83.33 ± 19.06
    91.20 ± 10.87
        3 months (11-13 weeks)
    87.82 ± 15.82
    85.12 ± 21.73
        15 months (59-62 weeks)
    96.21 ± 8.63
    88.33 ± 11.70
        24 months (95-97 weeks)
    94.44 ± 8.94
    93.06 ± 9.29
    Notes
    [19] - At baseline n=14, at T3 n=13, at T15 n=11 and at T24 n=12
    [20] - At baseline n=18, T3 n= 14, T15 n=15 and T24 n=12
    Statistical analysis title
    Role/school functioning (Self-report)-3 Month diff
    Statistical analysis description
    A mixed-effects model for repeated measures will be fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.181
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    8.92
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.42
         upper limit
    22.26
    Statistical analysis title
    Role/school functioning (Self-report)15 treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures will be fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.267
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    7.66
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.21
         upper limit
    21.52
    Statistical analysis title
    Role/school functioning (Self-report)24 month diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.328
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    6.97
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -7.39
         upper limit
    21.33
    Statistical analysis title
    Role/school functioning (Self-report) -treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.204
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    8.09
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.7
         upper limit
    20.89

    Secondary: CFQ - Vitality – Self Report

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    End point title
    CFQ - Vitality – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    14 [21]
    18 [22]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    50.60 ± 24.56
    63.89 ± 20.21
        3 Months (11-13 weeks)
    68.59 ± 17.06
    64.88 ± 21.48
        15 Months (59-62 weeks)
    74.24 ± 17.26
    66.67 ± 18.90
        24 Months (95-97 weeks)
    72.92 ± 18.84
    73.61 ± 16.60
    Notes
    [21] - At baseline n=14, T3 n=13, T15 n=11 and T24 n=12
    [22] - At baseline n=18, T3 n=14, T15 n=15 and T24 n=12
    Statistical analysis title
    Vitality (Self Report) - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.182
    Method
    Mixed models analysis
    Parameter type
    3 Month Treatment Difference
    Point estimate
    9.07
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.56
         upper limit
    22.69
    Statistical analysis title
    Vitality (Self Report) - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.373
    Method
    Mixed models analysis
    Parameter type
    15 Month Treatment Difference
    Point estimate
    5.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.88
         upper limit
    17.69
    Statistical analysis title
    Vitality (Self Report) - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.132
    Method
    Mixed models analysis
    Parameter type
    24 Month Treatment Difference
    Point estimate
    10.07
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.27
         upper limit
    23.41
    Statistical analysis title
    Vitality (Self Report) - Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.191
    Method
    Mixed models analysis
    Parameter type
    Treatment Difference
    Point estimate
    6.89
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.67
         upper limit
    17.45

    Secondary: CFQ - Emotional functioning – Self Report

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    End point title
    CFQ - Emotional functioning – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    53 [23]
    61 [24]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    78.8 ± 16.5
    76.7 ± 14.5
        3 Months (11-13 weeks)
    78.1 ± 15.1
    77.1 ± 14.8
        15 Months (59-62 weeks)
    80.8 ± 14.3
    79 ± 13.6
        24 Months (95-97 weeks)
    80.3 ± 14.1
    78.1 ± 13.5
    Notes
    [23] - At baseline n=53, T3 n=49, T15 n=50 and T24 n=44
    [24] - At baseline n=56, T3 n=49, T15 n=50 and T24 n=45
    Statistical analysis title
    Emotional functioning (Self-report) - 3 month diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.977
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    -0.07
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.06
         upper limit
    4.91
    Statistical analysis title
    Emotional functioning (Self-report) -15 month diff
    Statistical analysis description
    A mixed-effects model for repeated measure was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.589
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    -1.59
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -7.39
         upper limit
    4.22
    Statistical analysis title
    Emotional functioning (Self-report) -24 month diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.964
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    -0.14
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.19
         upper limit
    5.91
    Statistical analysis title
    Emotional functioning (Self-report) - Treat Diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.807
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    -0.53
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.78
         upper limit
    3.73

    Secondary: CFQ - Social functioning – Self Report

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    End point title
    CFQ - Social functioning – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    53 [25]
    61 [26]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    73 ± 17.6
    73.7 ± 15.3
        3 Months (11-13 weeks)
    75.5 ± 16.5
    72.2 ± 16.3
        15 Months (59-62 weeks)
    74.4 ± 18.1
    73.8 ± 15.5
        24 Weeks (95-97 weeks)
    77.8 ± 13.9
    73 ± 15.8
    Notes
    [25] - At baseline n=53, T3 n=49, T15 n=49 and T24 n=44
    [26] - At baseline n=61, T3 n=56 , T15 n=50 and T24 n=45
    Statistical analysis title
    Social functioning (Self-report) - 3 month diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.311
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    2.98
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.82
         upper limit
    8.78
    Statistical analysis title
    Social functioning (Self-report) - 15 month diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.498
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    2.11
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.03
         upper limit
    8.25
    Statistical analysis title
    Social functioning (Self-report) - 24 month diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.159
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    4.31
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.72
         upper limit
    10.34
    Statistical analysis title
    Social functioning (Self-report) - Treatment Diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. . The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.138
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    3.16
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.03
         upper limit
    7.36

    Secondary: CFQ - Body Image – Self Report

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    End point title
    CFQ - Body Image – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    52 [27]
    61 [28]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    90.4 ± 16.8
    86.3 ± 17.6
        3 Months (11-13 weeks)
    90 ± 16.2
    86.4 ± 16.4
        15 Months (59-62 weeks)
    88.2 ± 20
    86.4 ± 17.7
        24 Months (95-97 weeks)
    90.4 ± 12
    88.4 ± 18.3
    Notes
    [27] - At baseline n=52, T3 n= 49, T15 n=50 and T24 n=43
    [28] - At baseline n=61, T3 n=56, T15 n=50 and T24 n=45
    Statistical analysis title
    Body image (Self-report) - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    113
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.443
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    2.23
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.51
         upper limit
    7.97
    Statistical analysis title
    Body image (Self-report) - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    113
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.309
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    -4.01
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -11.78
         upper limit
    3.77
    Statistical analysis title
    Body image (Self-report) - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    113
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.986
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    -0.07
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -7.58
         upper limit
    7.45
    Statistical analysis title
    Body image (Self-report) - treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable.The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    113
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.931
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    0.2
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.43
         upper limit
    4.48

    Secondary: CFQ - Eating Problems – Self Report

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    End point title
    CFQ - Eating Problems – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    53 [29]
    61 [30]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    91 ± 16.5
    84.9 ± 19.7
        3 Months (11-13 weeks)
    86.5 ± 18.8
    82.1 ± 20.5
        15 Months (59-62 weeks)
    88.3 ± 17.4
    86.4 ± 17.3
        24 Months (95-97 weeks)
    92.6 ± 13.6
    87.4 ± 15.6
    Notes
    [29] - At baseline n=53, T3 n=49, T15 n=50 and T24 n=44
    [30] - At baseline n=61, T3 n=56, T15 n=50 and T24 n=45
    Statistical analysis title
    Eating problems (Self-report) - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.453
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    2.52
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.12
         upper limit
    9.16
    Statistical analysis title
    Eating problems (Self-report) - 15 month treat dif
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.903
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    -0.39
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.78
         upper limit
    6
    Statistical analysis title
    Eating problems (Self-report) -24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.31
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    3.67
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.47
         upper limit
    10.81
    Statistical analysis title
    Eating problems (Self-report) -treatment diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.506
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    1.66
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.27
         upper limit
    6.58

    Secondary: CFQ - Treatment Burden – Self Report

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    End point title
    CFQ - Treatment Burden – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    52 [31]
    61 [32]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    77.4 ± 21.7
    74.1 ± 18.5
        3 Months (11-13 weeks)
    71.9 ± 25.4
    70.7 ± 21.9
        15 Months (59-62 weeks)
    76.4 ± 18.7
    71.1 ± 20.5
        24 Months (95-97 weeks)
    77.3 ± 20.3
    74.8 ± 20.3
    Notes
    [31] - At baseline n=52, T3 n=49, T15 n=50 and T24 n=43
    [32] - At baseline n=61, T3 n=56, T15 n=50 and T24 n=45
    Statistical analysis title
    Treatment burden (Self-report) -3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    113
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.244
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    4.92
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.42
         upper limit
    13.27
    Statistical analysis title
    Treatment burden (Self-report)-15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    113
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.532
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    2.86
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.19
         upper limit
    11.92
    Statistical analysis title
    Treatment burden (Self-report)-24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    113
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.343
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    4.68
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.07
         upper limit
    14.43
    Statistical analysis title
    Treatment burden (Self-report)- Treatment diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    113
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.167
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    4.23
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.8
         upper limit
    10.27

    Secondary: CFQ - Health Perceptions – Self Report

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    End point title
    CFQ - Health Perceptions – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    14 [33]
    18 [34]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    65.87 ± 27.38
    70.99 ± 16.23
        3 Months (11-13 weeks)
    78.21 ± 20.84
    67.46 ± 27.38
        15 Months (59-62 weeks)
    80.81 ± 19.30
    71.11 ± 25.13
        24 Weeks (95-97 weeks)
    72.22 ± 24.39
    69.44 ± 20.17
    Notes
    [33] - At baseline n=14, T3 n=13, T15 n=11 and T24 n=12
    [34] - At baseline n=18, T3 n=14, T15 n= 15 and T24 n=12
    Statistical analysis title
    Health perceptions (Self-report)-3 month treat dif
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.045
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    18.41
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.4
         upper limit
    36.42
    Statistical analysis title
    Health perceptions (Self-report)-15 month diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.583
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    5.06
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -13.72
         upper limit
    23.84
    Statistical analysis title
    Health perceptions (Self-report)-24 month diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.544
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    6.33
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -14.89
         upper limit
    27.54
    Statistical analysis title
    Health perceptions (Self-report)-treatment diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    32
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.13
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    12.46
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.96
         upper limit
    28.88

    Secondary: CFQ - Weight – Self Report

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    End point title
    CFQ - Weight – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    14 [35]
    17 [36]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    85.71 ± 25.2
    78.43 ± 35.24
        3 Months (11-13 weeks)
    94.44 ± 19.25
    92.86 ± 19.30
        15 Months (59-62 weeks)
    93.94 ± 13.48
    84.44 ± 21.33
        24 Months (95-97 weeks)
    88.89 ± 25.95
    80.56 ± 26.43
    Notes
    [35] - At baseline n=14, T3 n= 12, T15 n=11 and T24 n=12
    [36] - At baseline n=17, T3 n=14 , T15 n=15, T24 n=12
    Statistical analysis title
    Weight (self report) - 3 Month treatment diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Oral antibiotic therapy v Intravenous (IV) antibiotics
    Number of subjects included in analysis
    31
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.725
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    2.51
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -12.06
         upper limit
    17.08
    Statistical analysis title
    Weight (self report) - 15 Month treatment diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    31
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.888
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    1.4
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -19.02
         upper limit
    21.83
    Statistical analysis title
    Weight (self report) - 24 Month treatment diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    31
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.842
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    2.68
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -24.8
         upper limit
    30.17
    Statistical analysis title
    Weight (self report) - treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    31
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.603
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    2.5
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -7.29
         upper limit
    12.29

    Secondary: CFQ - Respiratory Symptoms – Self Report

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    End point title
    CFQ - Respiratory Symptoms – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 Months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    53 [37]
    61 [38]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    67.0 ± 20.2
    70.5 ± 18.5
        3 Months (11-13 weeks)
    76.8 ± 19.8
    77 ± 15.9
        15 Months (59-62 weeks)
    80.4 ± 15.7
    78.4 ± 16
        24 Months (95-97 weeks)
    83.2 ± 13.4
    82.3 ± 13.4
    Notes
    [37] - At baseline n=53, T3 n=48, T15 n=49 and T24 n=44
    [38] - At baseline n=61, at T3 n=48 , at T15 n=50 and T24 n=44
    Statistical analysis title
    Resp Symp Self Report - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.606
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    1.79
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.06
         upper limit
    8.64
    Statistical analysis title
    Resp Symp Self Report -15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.374
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    2.82
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.44
         upper limit
    9.08
    Statistical analysis title
    Resp Symp Self Report -24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.53
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    2.08
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.47
         upper limit
    8.64
    Statistical analysis title
    Resp Symp Self Report -treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures wsa fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.344
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    2.27
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.47
         upper limit
    7.01

    Secondary: CFQ - Digestive Symptoms – Self Report

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    End point title
    CFQ - Digestive Symptoms – Self Report
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 Months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    53 [39]
    61 [40]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    78.4 ± 25.3
    74.5 ± 22.3
        3 Months (11-13 weeks)
    75.7 ± 29.1
    74.2 ± 24.8
        15 Months (59-62 weeks)
    80.1 ± 24.1
    80.2 ± 22.3
        24 Months (95-97 weeks)
    78.6 ± 23.3
    83.2 ± 17.2
    Notes
    [39] - At baseline n=53, T3 n=47, T15 n=48 and T24 n=42
    [40] - At baseline n=61, T3 n=56, T15 n=50 and T24 n=45
    Statistical analysis title
    Digestive (Self report) - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.551
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    2.72
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.3
         upper limit
    11.75
    Statistical analysis title
    Digestive (Self report) -15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.998
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    -0.01
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -9.95
         upper limit
    9.93
    Statistical analysis title
    Digestive (Self report) - treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.987
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    0.05
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.23
         upper limit
    6.33
    Statistical analysis title
    Digestive (Self report) - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    114
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.581
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    -2.49
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -11.44
         upper limit
    6.45

    Secondary: CFQ - Physical functioning – Parent/Carer

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    End point title
    CFQ - Physical functioning – Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 Months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    37 [41]
    42 [42]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    82.9 ± 18.3
    85.9 ± 14.3
        3 Months (11-13 weeks)
    90.1 ± 10.5
    84 ± 15.1
        15 Months (59-62 weeks)
    82.2 ± 20.9
    86.8 ± 15.6
        24 Months (95-97 weeks)
    82.8 ± 22
    89.2 ± 14.4
    Notes
    [41] - At baseline n=37, T3 n=38 , T15 n= 42, T24 n=32
    [42] - At baseline n=42, T3 n=39, T15 n=32 and T24 n=33
    Statistical analysis title
    Phys Fn - Parent/Carer - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.033
    Method
    Mixed models analysis
    Parameter type
    3 month treat diff
    Point estimate
    6.18
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.51
         upper limit
    11.85
    Statistical analysis title
    Phys Fn - Parent/Carer - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.223
    Method
    Mixed models analysis
    Parameter type
    15 month treat diff
    Point estimate
    -5.17
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -13.55
         upper limit
    3.22
    Statistical analysis title
    Phys Fn - Parent/Carer - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.342
    Method
    Mixed models analysis
    Parameter type
    24 month treat diff
    Point estimate
    -4.63
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -14.29
         upper limit
    5.03
    Statistical analysis title
    Phys Fn - Parent/Carer - treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.191
    Method
    Mixed models analysis
    Parameter type
    treat difference
    Point estimate
    3.62
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.85
         upper limit
    9.09

    Secondary: CFQ - Role/school functioning – Parent/Carer

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    End point title
    CFQ - Role/school functioning – Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    36 [43]
    42 [44]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    70.1 ± 27.1
    66.3 ± 21.4
        3 Months (11-13 weeks)
    73 ± 25.1
    66.7 ± 21
        15 Months (59-62 weeks)
    67.1 ± 29.3
    69.8 ± 22.3
        24 Months (95-97 weeks)
    72.2 ± 25.9
    78.1 ± 21.4
    Notes
    [43] - At baseline n= 36, T3 =37, T15 n=42 and T24 n=32
    [44] - At baseline n=42, T3 n=39, T15 n=32 and T24 n=33
    Statistical analysis title
    Role/School - P/C - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.238
    Method
    Mixed models analysis
    Parameter type
    3 month treat diff
    Point estimate
    5.47
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.69
         upper limit
    14.64
    Statistical analysis title
    Role/School - P/C - treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.845
    Method
    Mixed models analysis
    Parameter type
    treat diff
    Point estimate
    -0.67
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -7.49
         upper limit
    6.15
    Statistical analysis title
    Role/School - P/C - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.738
    Method
    Mixed models analysis
    Parameter type
    15 month treat diff
    Point estimate
    -1.47
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -10.22
         upper limit
    7.28
    Statistical analysis title
    Role/School - P/C - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.318
    Method
    Mixed models analysis
    Parameter type
    24 month treat diff
    Point estimate
    -5.06
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -15.09
         upper limit
    4.97

    Secondary: CFQ - Vitality– Parent/Carer

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    End point title
    CFQ - Vitality– Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    37 [45]
    41 [46]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    63.9 ± 17.4
    67 ± 16.8
        3 Months (11-13 weeks)
    67.1 ± 13.8
    62.2 ± 15.8
        15 Months (59-62 weeks)
    65.4 ± 18.1
    66.7 ± 18.4
        24 Months (95-97 weeks)
    66.5 ± 17.1
    68.3 ± 14.8
    Notes
    [45] - At baseline n=37, T3 n=38, T15 n=42 and T24 n=31
    [46] - At baseline n=41, T3 n=39 , T15 n=32 and T24 n=33
    Statistical analysis title
    Vitality - P/C - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.059
    Method
    Mixed models analysis
    Parameter type
    3 month treat diff
    Point estimate
    6.53
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.26
         upper limit
    13.32
    Statistical analysis title
    Vitality - P/C - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.923
    Method
    Mixed models analysis
    Parameter type
    15 month treat diff
    Point estimate
    -0.44
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -9.36
         upper limit
    8.48
    Statistical analysis title
    Vitality - P/C - treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable.The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.208
    Method
    Mixed models analysis
    Parameter type
    treat diff
    Point estimate
    3.7
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.11
         upper limit
    9.5
    Statistical analysis title
    Vitality - P/C - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.913
    Method
    Mixed models analysis
    Parameter type
    24 month treat diff
    Point estimate
    0.57
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -9.84
         upper limit
    10.98

    Secondary: CFQ - Emotional functioning – Parent/Carer

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    End point title
    CFQ - Emotional functioning – Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    36 [47]
    42 [48]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    77.2 ± 16.6
    80.3 ± 17
        3 Months (11-13 weeks)
    80.7 ± 14.2
    75.9 ± 18.1
        15 Months (59-62 weeks)
    79.9 ± 15.4
    79.6 ± 15.8
        24 Months (95-97 weeks)
    83.1 ± 14.1
    88.1 ± 10.5
    Notes
    [47] - At baseline n=36, T3 n=37, T15 n= 42, t24 n=32
    [48] - At baseline n=42, T3 n=39, T15 n=32 adn T24 n=33
    Statistical analysis title
    Emotional - P/C - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.069
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    5.97
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -0.47
         upper limit
    12.4
    Statistical analysis title
    Emotional - P/C - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.339
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    3.32
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.56
         upper limit
    10.2
    Statistical analysis title
    Emotional - P/C - treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.166
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    3.47
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.48
         upper limit
    8.41
    Statistical analysis title
    Emotional - P/C - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.668
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    -1.69
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -9.55
         upper limit
    6.16

    Secondary: CFQ - Body Image – Parent/Carer

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    End point title
    CFQ - Body Image – Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 Months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    36 [49]
    42 [50]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    75.3 ± 21.4
    72.5 ± 24.3
        3 Months (11-13 weeks)
    78.2 ± 22.5
    72.9 ± 24.4
        15 Months (59-62 weeks)
    71.8 ± 28
    76 ± 22.9
        24 months (95-97 weeks)
    71.9 ± 25.6
    82.5 ± 23.7
    Notes
    [49] - At baseline n=36, T3 n=36 , T15 n=42 and T24 n=32
    [50] - At baseline n=42, T3 n=39, T15 n=32, and T24 n=33
    Statistical analysis title
    Body Image - P/C - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Oral antibiotic therapy v Intravenous (IV) antibiotics
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.555
    Method
    Mixed models analysis
    Parameter type
    3 month treat diff
    Point estimate
    2.66
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.28
         upper limit
    11.59
    Statistical analysis title
    Body Image - P/C - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.922
    Method
    Mixed models analysis
    Parameter type
    15 month treat diff
    Point estimate
    -0.56
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -12.03
         upper limit
    10.9
    Statistical analysis title
    Body Image - P/C - treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.644
    Method
    Mixed models analysis
    Parameter type
    treat diff
    Point estimate
    1.94
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.4
         upper limit
    10.28
    Statistical analysis title
    Body Image - P/C - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.679
    Method
    Mixed models analysis
    Parameter type
    24 month treat diff
    Point estimate
    2.58
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -9.81
         upper limit
    14.97

    Secondary: CFQ - Eating Problems– Parent/Carer

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    End point title
    CFQ - Eating Problems– Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    37 [51]
    41 [52]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    77 ± 29.7
    78.9 ± 25.3
        3 Months (11-13 weeks)
    78.9 ± 26.7
    72.6 ± 22.8
        15 Months (59-62 weeks)
    77.8 ± 27.9
    78.6 ± 22.5
        24 Months (95-97 weeks)
    78.1 ± 24.1
    82.3 ± 19.4
    Notes
    [51] - Baseline n=37, T3 n=34 , T15 n=39 and T24 n=32
    [52] - At baseline n=41, T3 n=39, T15 n=32 and T24 n=32
    Statistical analysis title
    Eating Problem P/C - 3 Month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.3
    Method
    Mixed models analysis
    Parameter type
    3 month treatment difference
    Point estimate
    4.6
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.19
         upper limit
    13.39
    Statistical analysis title
    Eating Problem P/C - 15 Month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.174
    Method
    Mixed models analysis
    Parameter type
    15 month treatment difference
    Point estimate
    6.15
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -2.78
         upper limit
    15.08
    Statistical analysis title
    Eating Problem P/C - 24 Month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.732
    Method
    Mixed models analysis
    Parameter type
    24 month treatment difference
    Point estimate
    -1.94
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -13.18
         upper limit
    9.31
    Statistical analysis title
    Eating Problem P/C - Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable . The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.138
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    4.05
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -1.33
         upper limit
    9.44

    Secondary: CFQ - Treatment Burden – Parent/Carer

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    End point title
    CFQ - Treatment Burden – Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    37 [53]
    42 [54]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    57.8 ± 20.8
    55.4 ± 23.4
        3 Months (11-13 weeks)
    59.3 ± 24.3
    51.6 ± 23.8
        15 Months (59-62 week)
    57.7 ± 23.6
    54.2 ± 20.9
        24 Months (95-97 weeks)
    58.3 ± 22.9
    57.9 ± 21.8
    Notes
    [53] - At baseline n=37, T3 n=37, T15 n=42, T24 n=32
    [54] - At baseline n=42, T3 n=39, T15 n=32, T24 n=33
    Statistical analysis title
    Treatment Burden P/C - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.718
    Method
    Mixed models analysis
    Parameter type
    3 month treat diff
    Point estimate
    1.99
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -8.94
         upper limit
    12.92
    Statistical analysis title
    Treatment Burden P/C - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.96
    Method
    Mixed models analysis
    Parameter type
    15 month treat diff
    Point estimate
    -0.28
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -11.25
         upper limit
    10.69
    Statistical analysis title
    Treatment Burden P/C - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.966
    Method
    Mixed models analysis
    Parameter type
    24 month treat diff
    Point estimate
    0.23
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -10.12
         upper limit
    10.57
    Statistical analysis title
    Treatment Burden P/C - treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.862
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    0.68
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -7.08
         upper limit
    8.44

    Secondary: CFQ - Health Perceptions – Parent/Carer

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    End point title
    CFQ - Health Perceptions – Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    36 [55]
    42 [56]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    71.6 ± 21
    70.1 ± 18.3
        3 Months (11-13 weeks)
    69.9 ± 20
    70.1 ± 18.4
        15 Months (59-62 weeks)
    67.9 ± 17.6
    74.7 ± 15.5
        24 Months (95-97 weeks)
    72.2 ± 23.1
    79.8 ± 17.7
    Notes
    [55] - At baseline n=36, T3 n=36, T15 n=42, T24 n=32
    [56] - At bseline n=42, T3 n=39, T15 n=32, T24 n=33
    Statistical analysis title
    Health Perceptions P/C - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.988
    Method
    Mixed models analysis
    Parameter type
    3 month treat diff
    Point estimate
    -0.07
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -9.37
         upper limit
    9.23
    Statistical analysis title
    Health Perceptions P/C - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.07
    Method
    Mixed models analysis
    Parameter type
    15 month treat diff
    Point estimate
    -7.25
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -15.09
         upper limit
    0.6
    Statistical analysis title
    Health Perceptions P/C - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.297
    Method
    Mixed models analysis
    Parameter type
    24 month treat diff
    Point estimate
    -6.76
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -19.6
         upper limit
    6.07
    Statistical analysis title
    Health Perceptions P/C - treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.115
    Method
    Mixed models analysis
    Parameter type
    treat diff
    Point estimate
    -4.39
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -9.88
         upper limit
    1.1

    Secondary: CFQ - Weight – Parent/Carer

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    End point title
    CFQ - Weight – Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    37 [57]
    42 [58]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    64 ± 31.8
    69.8 ± 32.8
        3 Months (11-13 weeks)
    70.3 ± 33.1
    62.2 ± 30.6
        15 Months (59-62 weeks)
    64.2 ± 35.3
    69.8 ± 30.9
        24 Months (95-97 weeks)
    64.6 ± 35.9
    69.7 ± 36.7
    Notes
    [57] - At baseline n=37, T3 n=37, T15 n=41 and T24 n=32
    [58] - At baseline n=42, T3 n=37, T15 n=32 and T24 n=33
    Statistical analysis title
    Weight - P/C - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.294
    Method
    Mixed models analysis
    Parameter type
    3 month treat diff
    Point estimate
    7.66
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -6.78
         upper limit
    22.1
    Statistical analysis title
    Weight - P/C - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.886
    Method
    Mixed models analysis
    Parameter type
    15 month treat diff
    Point estimate
    -1.19
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -17.63
         upper limit
    15.26
    Statistical analysis title
    Weight - P/C - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.492
    Method
    Mixed models analysis
    Parameter type
    24 month treat diff
    Point estimate
    7.03
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -13.27
         upper limit
    27.33
    Statistical analysis title
    Weight - P/C - treatment difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.247
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    6.37
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.5
         upper limit
    17.25

    Secondary: CFQ - Respiratory Symptoms– Parent/Carer

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    End point title
    CFQ - Respiratory Symptoms– Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    37 [59]
    42 [60]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    68.6 ± 20.9
    73.5 ± 21.9
        3 Months (11-13 weeks)
    82.1 ± 18.1
    79.1 ± 19.5
        15 Months (59-62 weeks)
    76.1 ± 18.2
    82.6 ± 14.9
        24 Months (95-97 weeks)
    78.3 ± 20.2
    82 ± 16.3
    Notes
    [59] - At baseline n=37, T3 n=37, T15 n=41 and T24 n=32
    [60] - At baseline n=42, T3 n=39, T15 n=32 and T24 n=33
    Statistical analysis title
    Resp Symp P/C - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.39
    Method
    Mixed models analysis
    Parameter type
    3 month treat diff
    Point estimate
    4.11
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -5.36
         upper limit
    13.58
    Statistical analysis title
    Resp Symp P/C - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.432
    Method
    Mixed models analysis
    Parameter type
    15 month treat diff
    Point estimate
    -3.33
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -11.71
         upper limit
    5.06
    Statistical analysis title
    Resp Symp P/C - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.728
    Method
    Mixed models analysis
    Parameter type
    24 month treat diff
    Point estimate
    -2.11
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -14.15
         upper limit
    9.93
    Statistical analysis title
    Resp Symp P/C - treament difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Oral antibiotic therapy v Intravenous (IV) antibiotics
    Number of subjects included in analysis
    79
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.885
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    -0.47
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -7.01
         upper limit
    6.06

    Secondary: CFQ - Digestive symptoms – Parent/Carer

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    End point title
    CFQ - Digestive symptoms – Parent/Carer
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 24 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    37 [61]
    41 [62]
    Units: CFQ Score
    arithmetic mean (standard deviation)
        Baseline
    70.9 ± 22.6
    74 ± 19.5
        3 Months (11-13 weeks)
    76.8 ± 20
    74.5 ± 13.2
        15 months (59-62 weeks)
    77.2 ± 18.7
    73.6 ± 20.5
        24 Weeks (95-97 weeks)
    76.4 ± 16.2
    79.5 ± 17.4
    Notes
    [61] - At baseline n=37, T3 n=34 , T15 n=39 and T24 n=32
    [62] - At baseline n=41, T3 n=39, T15 n=32 and T24 n=32
    Statistical analysis title
    Digestive - P/C - 3 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.474
    Method
    Mixed models analysis
    Parameter type
    3 month treat diff
    Point estimate
    2.66
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.71
         upper limit
    10.03
    Statistical analysis title
    Digestive - P/C - 15 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.405
    Method
    Mixed models analysis
    Parameter type
    15 month treat diff
    Point estimate
    3.54
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -4.9
         upper limit
    11.98
    Statistical analysis title
    Digestive - P/C - 24 month treat diff
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group, time-point as a categorical variable and an interaction term (treatment group*time). The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.725
    Method
    Mixed models analysis
    Parameter type
    24 month treat diff
    Point estimate
    1.75
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -8.12
         upper limit
    11.62
    Statistical analysis title
    Digestive - P/C - Treatment Difference
    Statistical analysis description
    A mixed-effects model for repeated measures was fitted, with an unstructured covariance matrix. The baseline measurement of the domain was fitted as a covariate along with treatment group and time-point as a categorical variable. The following was reported: mean (SD) for each treatment group; mean (95% CI) difference between treatment groups (derived from the model); and a p-value of the treatment effect
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    78
    Analysis specification
    Post-hoc
    Analysis type
    superiority
    P-value
    = 0.389
    Method
    Mixed models analysis
    Parameter type
    treatment difference
    Point estimate
    2.61
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -3.4
         upper limit
    8.61

    Secondary: Number of patients with at least one positive result of MRSA by 3 months

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    End point title
    Number of patients with at least one positive result of MRSA by 3 months
    End point description
    No analysis was possible due to there being 0 events in the IV group.
    End point type
    Secondary
    End point timeframe
    Baseline to three months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    136
    145
    Units: Subjects
        0 Culture
    136
    144
        >= 1 culture
    0
    1
    No statistical analyses for this end point

    Secondary: Number of patients with at least one positive result of Burkholderia cepacia complex (BC) by 3 months

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    End point title
    Number of patients with at least one positive result of Burkholderia cepacia complex (BC) by 3 months
    End point description
    No analysis was possible due to there being 0 events in the Oral group.
    End point type
    Secondary
    End point timeframe
    Baseline to three months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    136
    144
    Units: Subjects
        0 culture
    135
    144
        >=1 culture
    1
    0
    No statistical analyses for this end point

    Secondary: Number of patients with at least one positive result of Candida by 3 months

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    End point title
    Number of patients with at least one positive result of Candida by 3 months
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 3 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    137
    146
    Units: Subjects
        0 culture
    111
    119
        >=1 culture
    26
    27
    Statistical analysis title
    Secondary Outcome - Relative Risk
    Statistical analysis description
    For each microorganism, the number and percentage with at least one positive result were presented split by treatment arm, and a relative risk and 95% confidence interval calculated. Chi-squared test (or if necessary Fisher’s exact test) was used to test for a difference between treatment groups.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    283
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.917
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    1.03
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.63
         upper limit
    1.67

    Secondary: Number of patients with at least one positive result of Aspergillus by 3 months

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    End point title
    Number of patients with at least one positive result of Aspergillus by 3 months
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 3 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    136
    144
    Units: Subjects
        0 culture
    130
    139
        >=1 culture
    6
    5
    Statistical analysis title
    Secondary Outcome - Relative Risk
    Statistical analysis description
    For each microorganism, the number and percentage with at least one positive result waspresented split by treatment arm, and a relative risk and 95% confidence interval calculated. Chi-squared tests (or if necessary Fisher’s exact test) was used to test for differences between treatment groups.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    280
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.686
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    1.27
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.4
         upper limit
    4.07

    Secondary: Number of patients with at least one positive result of MRSA by 15 months

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    End point title
    Number of patients with at least one positive result of MRSA by 15 months
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 15 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    135
    140
    Units: Subjects
        0 culture
    131
    138
        >=1 culture
    4
    2
    Statistical analysis title
    Secondary Outcome - Relative Risk
    Statistical analysis description
    For each microorganism, the number and percentage with at least one positive result was presented split by treatment arm, and a relative risk and 95% confidence interval calculated. Chi-squared tests (or if necessary Fisher’s exact test) was used to test for differences between treatment groups.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    275
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.441
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    2.07
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.39
         upper limit
    11.14

    Secondary: Number of patients with at least one positive result of Burkholderia cepacia complex (BC) by 15 months

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    End point title
    Number of patients with at least one positive result of Burkholderia cepacia complex (BC) by 15 months
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline to 15 months
    End point values
    Intravenous (IV) antibiotics Oral antibiotic therapy
    Number of subjects analysed
    135
    139
    Units: Subjects
        0 culture
    133
    135
        >= 1 culture
    2
    4
    Statistical analysis title
    Secondary Outcome - Relative Risk
    Statistical analysis description
    For each microorganism, the number and percentage with at least one positive result was presented split by treatment arm, and a relative risk and 95% confidence interval calculated. Chi-squared tests (or if necessary Fisher’s exact test) was used to test for differences between treatment groups.
    Comparison groups
    Intravenous (IV) antibiotics v Oral antibiotic therapy
    Number of subjects included in analysis
    274
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.684
    Method
    Chi-squared
    Parameter type
    Risk ratio (RR)
    Point estimate
    0.51
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.1
         upper limit
    2.76

    Secondary: Number of patients with at least one positive result of Candida by 15 months

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    End point title
    Number of patients with at least one positive result of Candida by 15 months
    End point description