Findings

What do systematic reviews say?

100 questions · 100 findings · 3 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 30 · 30%GRADE not reported 4 · 4%Very low 5 · 5%Low 26 · 26%Moderate 31 · 31%High 4 · 4%

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

FEV1 % predicted: absolute 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
1 study · 204 participants
Low

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

Time to first pulmonary exacerbation · 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

Survival · 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 · 62 participants
Moderate

Summary of findings ‐ monotherapy: lumacaftor vs placebo

Quality of life: CFQ‐R respiratory domain: absolute 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
1 study · 85 participants
Low

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

Quality of life: total score · 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

Quality of life: respiratory domain · 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: lumacaftor plus ivacaftor vs placebo

FEV1 % predicted: absolute 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
1 study · 59 participants
Moderate

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 ‐ dual therapy: lumacaftor plus ivacaftor vs placebo

Time to first pulmonary exacerbation · 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

Survival · 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
9 studies · 1132 participants
Low

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

Quality of life: total score · 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

Quality of life: CFQ‐R respiratory domain: absolute 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 · 946 participants
Low

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 ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone

FEV1 % predicted: absolute 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 ‐ 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 ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone

Time to first pulmonary exacerbation · 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 · 506 participants
Moderate

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

Survival · 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