Cochrane · systematic review · CD009841
Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis
Skilton, M, Krishan, A, Patel, S, et al. · Cochrane Database of Systematic Reviews · 2019 · Cochrane Library
Cystic Fibrosis and Genetic DisordersAMSTAR 2: not assessed
SoF tableOnly the outcomes this review put in its Summary of Findings table are listed (the Cochrane Handbook caps them at about seven per comparison). Its other analyses are not shown here.
Questions
21
current version
GRADE rated
14
No studies
4
Included studies
NR
No studies 4 · 19%Ungraded 3 · 14.3%Very low 0 · 0%Low 4 · 19%Moderate 10 · 47.6%High 0 · 0%
Ungraded means an effect was reported without a GRADE rating; it is not a gap. No studies means the review found nothing to answer the question. The six categories are a display order, not a score.
Questions and results
Summary of findings ‐ ivacaftor compared with placebo for cystic fibrosis with the F508del CFTR mutation
Population: adults and children with cystic fibrosis and with the F508del CFTR mutation
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 1 study · 140 participants | Ungraded |
QoL Total score Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | NR | No studies |
QoL (CFQ‐R) Respiratory domain Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 1 study · 140 participants | Moderate Downgraded: risk of bias, imprecision |
FEV1 % predicted Relative change from baseline Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 1 study · 140 participants | Moderate Downgraded: risk of bias, imprecision |
FEV1 % predicted Absolute change from baseline Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 1 study · 140 participants | Moderate Downgraded: risk of bias, imprecision |
Adverse events Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 1 study · 140 participants | Low Downgraded: risk of bias, imprecision |
Time to first pulmonary exacerbation Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ ivacaftor compared with placebo for cystic fibrosis with at least one G551D CFTR mutation
Population: adults and children with cystic fibrosis and with at least one G551D CFTR mutation
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 3 studies · 238 participants | Ungraded |
QoL Total score Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | NR | No studies |
QoL (CFQ‐R) Respiratory domain Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 3 studies · 222 participants | Moderate Downgraded: risk of bias, imprecision, inconsistency |
FEV1 % predicted Relative change from baseline Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 3 studies · 232 participants | Moderate Downgraded: risk of bias, imprecision, inconsistency |
FEV1 L and % predicted Absolute change from baseline Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 3 studies · 232 participants | Low Downgraded: risk of bias, imprecision, inconsistency |
Adverse events Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 3 studies · 232 participants | Low Downgraded: risk of bias, imprecision, inconsistency |
Time to first pulmonary exacerbation Summary of findings ‐ ivacaftor vs placebo | HR 0.46 [0.29, 0.73] | 1 study · 161 participants | Moderate Downgraded: risk of bias, imprecision, inconsistency |
Summary of findings ‐ ivacaftor compared with placebo for cystic fibrosis with at least one R117H CFTR mutation
Population: adults and children with cystic fibrosis and with at least one R117H CFTR mutation
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 1 study · 69 participants | Ungraded |
QoL Total score Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | NR | No studies |
QoL (CFQ‐R) Respiratory domain Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 1 study · 69 participants | Moderate Downgraded: risk of bias, imprecision |
FEV1 % predicted Relative change from baseline Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 1 study · 69 participants | Moderate Downgraded: risk of bias, imprecision |
FEV1 L and % predicted Absolute change from baseline Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 1 study · 69 participants | Moderate Downgraded: risk of bias, imprecision |
Adverse events Summary of findings ‐ ivacaftor vs placebo | no estimate extracted | 1 study · 69 participants | Low Downgraded: risk of bias, imprecision |
Time to first pulmonary exacerbation Summary of findings ‐ ivacaftor vs placebo | HR 0.93 [0.42, 2.08] | 1 study · 69 participants | Moderate Downgraded: risk of bias, imprecision |