Findings

What do systematic reviews say?

11 questions · 11 findings · 1 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 0 · 0%GRADE not reported 0 · 0%Very low 2 · 18.2%Low 4 · 36.4%Moderate 4 · 36.4%High 1 · 9.1%

Summary of findings ‐ monotherapy: lumacaftor vs placebo

Adverse events · 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
2 studies · 115 participants
Very low

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

Adverse events · 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
1 study · 61 participants
Low

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

Adverse events · 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

Adverse events · 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 ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo

Adverse events · 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
2 studies · 1108 participants
High

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

Adverse events · 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
1 study · 204 participants
Low

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

Adverse events · 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
4 studies · 634 participants
Moderate

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

Adverse events · 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 ‐ monotherapy: cavosonstat vs placebo

Adverse events: occurring in at least 10% of cavosonstat treated participants · 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
1 study · 26 participants
Very low

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

Adverse events: most commonly occurring events (occurring in at least 10% of participants) · 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
8 studies · 1057 participants
Moderate

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

Adverse events ‐ treatment emergent · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor showed no clear difference in adverse events: number of participants with treatment‐emergent… compared with control (ivacaftor alone or tezacaftor plus ivacaftor) (moderate certainty).

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
OR 1.04 [0.53, 2.04]
1 study · 258 participants
Moderate