Findings

What do systematic reviews say?

19 questions · 19 findings · 5 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 6 · 31.6%GRADE not reported 1 · 5.3%Very low 0 · 0%Low 3 · 15.8%Moderate 6 · 31.6%High 3 · 15.8%

Ataluren vs placebo

FEV1 % predicted: relative change from baseline · adults and children with CF with at least one class I mutation (PTC)

1 finding from 1 review

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 203 participants
High

Summary of findings ‐ ivacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis and with at least one G551D CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
3 studies · 232 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis and with at least one R117H CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 69 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis and with the F508del CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 140 participants
Moderate

Summary of findings ‐ monotherapy: cavosonstat vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
3 studies · 1134 participants
High

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
5 studies · 944 participants
Low

Summary of findings ‐ monotherapy: lumacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control

FEV1 % predicted: relative change from baseline · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
6 participants
GRADE not reported

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control

FEV1 % predicted: relative change from baseline · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
3 studies · 603 participants
Moderate

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

FEV1 % predicted: relative change from baseline · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype

The review found no eligible studies for this question.

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults with cystic fibrosis and with the F508del/MF genotype

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 55 participants
Low

Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults with cystic fibrosis and with the F508del/MF genotype

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 76 participants
Low

Summary of findings: tobramycin for inhalation powder vs tobramycin

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 517 participants
Moderate

Summary of findings: levofloxacin for inhalation solution vs tobramycin

FEV1 % predicted: relative change from baseline · adults and children aged over 12 with CF and P aeruginosa

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 282 participants
High

Oral ciprofloxacin and inhaled colistin vs inhaled tobramycin (TNS) plus oral ciprofloxacin

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

The review found no eligible studies for this question.

1 finding from 1 review

Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings: aztreonam lysine for inhalation vs tobramycin

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 268 participants
Moderate