Findings

What do systematic reviews say?

84 questions · 84 findings · 2 reviews · Headline outcomes only

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No studies found 24 · 28.6%GRADE not reported 4 · 4.8%Very low 0 · 0%Low 22 · 26.2%Moderate 30 · 35.7%High 4 · 4.8%

Summary of findings ‐ ivacaftor vs placebo

Survival · 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
GRADE not reported

Summary of findings ‐ ivacaftor vs placebo

QoL (CFQ‐R) Respiratory domain · 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 · 222 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 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 L and % predicted Absolute 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
Low

Summary of findings ‐ ivacaftor vs placebo

Adverse events · 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
Low

Summary of findings ‐ ivacaftor vs placebo

Time to first pulmonary exacerbation · adults and children with cystic fibrosis and with at least one G551D CFTR mutation

Summary of findings ‐ ivacaftor probably leads to lower time to first pulmonary exacerbation compared with placebo (moderate certainty).

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
HR 0.46 [0.29, 0.73]
1 study · 161 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

Survival · 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
GRADE not reported

Summary of findings ‐ ivacaftor vs placebo

QoL · adults and children with cystic fibrosis and with at least one R117H CFTR mutation

The review found no eligible studies for this question.

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
Studies not reported
No studies found

Summary of findings ‐ ivacaftor vs placebo

QoL (CFQ‐R) Respiratory domain · 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 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 L and % predicted Absolute 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

QoL · adults and children with cystic fibrosis and with the F508del CFTR mutation

The review found no eligible studies for this question.

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
Studies not reported
No studies found

Summary of findings ‐ ivacaftor vs placebo

Adverse events · 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
Low

Summary of findings ‐ ivacaftor vs placebo

Time to first pulmonary exacerbation · adults and children with cystic fibrosis and with at least one R117H CFTR mutation

Summary of findings ‐ ivacaftor showed no clear difference in time to first pulmonary exacerbation compared with placebo (moderate certainty).

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
HR 0.93 [0.42, 2.08]
1 study · 69 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

QoL (CFQ‐R) Respiratory domain · 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 ‐ 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 ‐ ivacaftor vs placebo

FEV1 % predicted: absolute 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 ‐ ivacaftor vs placebo

Adverse events · 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
Low

Summary of findings ‐ ivacaftor vs placebo

Time to first pulmonary exacerbation · adults and children with cystic fibrosis and with the F508del CFTR mutation

The review found no eligible studies for this question.

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
Studies not reported
No studies found

Summary of findings ‐ ivacaftor vs placebo

Survival · 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 · 238 participants
GRADE not reported