Cochrane · systematic review · CD010966
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)
Heneghan, M, Southern, KW, Murphy, J, et al. · Cochrane Database of Systematic Reviews · 2023 · Cochrane Library
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
77
current version
GRADE rated
50
No studies
26
Included studies
NR
No studies 26 · 33.8%Ungraded 1 · 1.3%Very low 5 · 6.5%Low 20 · 26%Moderate 21 · 27.3%High 4 · 5.2%
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
Current version onlySummary of findings ‐ monotherapy: lumacaftor compared to placebo for cystic fibrosis
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | 2 studies · 147 participants | Low Downgraded: risk of bias, imprecision |
Quality of life ‐ total score Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | NR | No studies |
Quality of life ‐ CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | 1 study · 85 participants | Low Downgraded: risk of bias, imprecision |
FEV1 % predicted: relative change from baseline Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | 1 study · 61 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Adverse events Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | 2 studies · 115 participants | Very low Downgraded: risk of bias, imprecision |
Time to first pulmonary exacerbation Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ monotherapy: cavosonstat compared to placebo for cystic fibrosis
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Very low Downgraded: risk of bias |
Quality of life: total score Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | 1 study · 24 participants | Very low Downgraded: risk of bias |
FEV1 % predicted: relative change from baseline Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Very low Downgraded: risk of bias |
Adverse events: occurring in at least 10% of cavosonstat‐treated participants Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Very low Downgraded: risk of bias |
Time to first pulmonary exacerbation Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) compared with placebo for cystic fibrosis (short term)
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1108 participants | High |
Quality of life ‐ (EuroQol) EQ‐5D‐3L Index Score (total score): absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1061 participants | Moderate Downgraded: risk of bias |
Quality of life ‐ CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 3 studies · 1139 participants | Moderate Downgraded: risk of bias |
FEV1 % predicted: relative change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 3 studies · 1134 participants | High |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 3 studies · 1134 participants | Moderate Downgraded: risk of bias |
Adverse events Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1108 participants | High |
Time to first pulmonary exacerbation Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1108 participants | Moderate Downgraded: risk of bias |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) compared with placebo for cystic fibrosis (short term)
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Moderate Downgraded: indirectness |
Quality of life ‐ total score Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | NR | No studies |
Quality of life ‐ CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Low Downgraded: indirectness |
FEV1 % predicted: relative change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Low Downgraded: indirectness |
Adverse events Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Low Downgraded: risk of bias, imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor compared with placebo for cystic fibrosis (immediate term)
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | 1 study · 62 participants | Moderate Downgraded: imprecision, indirectness |
Quality of life: total score Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
Quality of life: respiratory domain Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: relative change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | 1 study · 59 participants | Moderate Downgraded: imprecision, indirectness |
Adverse events Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | 1 study · 61 participants | Low Downgraded: imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor compared with placebo or ivacaftor alone for cystic fibrosis
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 9 studies · 1132 participants | Low Downgraded: indirectness |
Quality of life: total score Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 5 studies · 946 participants | Low Downgraded: indirectness |
FEV1 % predicted: relative change from baseline Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 5 studies · 944 participants | Low Downgraded: indirectness |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 5 studies · 944 participants | Low Downgraded: indirectness |
Adverse events: most commonly occurring events (occurring in at least 10% of participants) Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 8 studies · 1057 participants | Moderate Downgraded: indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 1 study · 506 participants | Moderate Downgraded: indirectness |
Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor compared to control for cystic fibrosis
Population: adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | 4 studies · 634 participants | Moderate Downgraded: indirectness |
Quality of life: total score Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | 1 study · 382 participants | Moderate Downgraded: indirectness |
FEV1 (% predicted): relative change from baseline Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | 6 participants | Ungraded |
FEV1 (% predicted): absolute change from baseline Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | 1 study · 382 participants | Moderate Downgraded: indirectness |
Adverse events Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | 4 studies · 634 participants | Moderate Downgraded: indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | NR | No studies |
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor compared to control for cystic fibrosis
Population: adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | 3 studies · 603 participants | High |
Quality of life: total score Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | 3 studies · 599 participants | Moderate Downgraded: indirectness |
FEV1 (% predicted): relative change from baseline Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | 3 studies · 603 participants | Moderate Downgraded: indirectness |
FEV1 (% predicted): absolute change from baseline Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | 2 studies · 578 participants | Moderate Downgraded: indirectness |
Adverse events Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | 3 studies · 603 participants | Moderate Downgraded: indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control | no estimate extracted | NR | No studies |
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor compared to control (ivacaftor alone or tezacaftor plus ivacaftor) for cystic fibrosis
Population: adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) | no estimate extracted | 1 study · 258 participants | Moderate Downgraded: indirectness |
Quality of life: total score Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) | no estimate extracted | NR | No studies |
FEV1 (% predicted): relative change from baseline Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) | no estimate extracted | NR | No studies |
FEV1 (% predicted): absolute change from baseline Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) | no estimate extracted | 1 study · 258 participants | Moderate Downgraded: indirectness |
Adverse events: number of participants with treatment‐emergent adverse events Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) | OR 1.04 [0.53, 2.04] | 1 study · 258 participants | Moderate Downgraded: indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) | no estimate extracted | NR | No studies |
Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor compared to placebo for cystic fibrosis
Population: adults with cystic fibrosis and with the F508del/MF genotype
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | 1 study · 55 participants | Low Downgraded: imprecision, indirectness |
Quality of life: total score Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | 1 study · 55 participants | Low Downgraded: imprecision, indirectness |
FEV1 (% predicted): relative change from baseline Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | 1 study · 55 participants | Low Downgraded: imprecision, indirectness |
FEV1 (% predicted): absolute change from baseline Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | 1 study · 55 participants | Low Downgraded: imprecision, indirectness |
Adverse events Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | 1 study · 55 participants | Low Downgraded: imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor compared to placebo for cystic fibrosis
Population: adults with cystic fibrosis and with the F508del/MF genotype
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | 1 study · 76 participants | Low Downgraded: risk of bias, indirectness |
Quality of life: total score Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | 1 study · 76 participants | Low Downgraded: risk of bias, indirectness |
FEV1 (% predicted): relative change from baseline Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | 1 study · 76 participants | Low Downgraded: risk of bias, indirectness |
FEV1 (% predicted): absolute change from baseline Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | 1 study · 76 participants | Low Downgraded: risk of bias, indirectness |
Adverse events Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | 1 study · 76 participants | Low Downgraded: risk of bias, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ monotherapy: lumacaftor compared to placebo for cystic fibrosis
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | 2 studies · 147 participants | Low Downgraded: risk of bias, imprecision, indirectness |
Quality of life ‐ total score Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | NR | No studies |
Quality of life ‐ CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | 1 study · 85 participants | Low Downgraded: risk of bias, imprecision, indirectness |
FEV1 % predicted: relative change from baseline Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | 1 study · 61 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Adverse events Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | 2 studies · 115 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ monotherapy: lumacaftor vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ monotherapy: cavosonstat compared to placebo for cystic fibrosis
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Quality of life: total score Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
FEV1 % predicted: relative change from baseline Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Adverse events: occurring in at least 10% of cavosonstat treated participants Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ monotherapy: cavosonstat vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) compared with placebo for cystic fibrosis (short term)
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1108 participants | High |
Quality of life ‐ (EuroQol) EQ‐5D‐3L Index Score (total score): absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1061 participants | Moderate Downgraded: risk of bias |
Quality of life ‐ CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1076 participants | Moderate Downgraded: risk of bias |
FEV1 % predicted: relative change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1072 participants | High |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1072 participants | Moderate Downgraded: risk of bias |
Adverse events Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1108 participants | High |
Time to first pulmonary exacerbation Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1108 participants | Moderate Downgraded: risk of bias |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) compared with placebo for cystic fibrosis (short term)
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Quality of life ‐ total score Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | NR | No studies |
Quality of life ‐ CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Low Downgraded: risk of bias, imprecision, indirectness |
FEV1 % predicted: relative change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Low Downgraded: risk of bias, imprecision, indirectness |
Adverse events Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Low Downgraded: risk of bias, imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor compared with placebo for cystic fibrosis (immediate term)
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | 1 study · 62 participants | Moderate Downgraded: imprecision, indirectness |
Quality of life: total score Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
Quality of life: respiratory domain Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: relative change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | 1 study · 59 participants | Moderate Downgraded: imprecision, indirectness |
Adverse events Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | 1 study · 61 participants | Low Downgraded: imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor compared with placebo or ivacaftor alone for cystic fibrosis
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 2 studies · 522 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Quality of life: total score Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 2 studies · 522 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
FEV1 % predicted: relative change from baseline Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 2 studies · 522 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 2 studies · 522 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Adverse events: most commonly occurring events (occurring in at least 10% of participants) Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 2 studies · 527 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 1 study · 504 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/VX‐561 compared to control for cystic fibrosis
Population: adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 2 studies · 129 participants | High |
Quality of life: total score Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 2 studies · 129 participants | Moderate Downgraded: indirectness |
FEV1 (% predicted): relative change from baseline Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 1 study · 117 participants | Moderate Downgraded: indirectness |
FEV1 (% predicted): absolute change from baseline Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 1 study · 12 participants | Moderate Downgraded: indirectness |
Adverse events Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 2 studies · 129 participants | Moderate Downgraded: indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | NR | No studies |
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/VX‐561 compared to control for cystic fibrosis
Population: adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 3 studies · 603 participants | High |
Quality of life: total score Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain absolute change from baseline Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 3 studies · 599 participants | Moderate Downgraded: indirectness |
FEV1 (% predicted): relative change from baseline Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 3 studies · 603 participants | Moderate Downgraded: indirectness |
FEV1 (% predicted): absolute change from baseline Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 2 studies · 510 participants | Ungraded |
Adverse events Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 3 studies · 603 participants | Moderate Downgraded: indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/VX‐561 vs control | no estimate extracted | 1 study · 403 participants | Moderate Downgraded: indirectness |
Summary of findings ‐ Lumacaftor monotherapy compared to placebo
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ Lumacaftor monotherapy vs placebo | no estimate extracted | 2 studies · 147 participants | Low Downgraded: risk of bias, imprecision, indirectness |
Quality of life ‐ total score Summary of findings ‐ Lumacaftor monotherapy vs placebo | no estimate extracted | NR | No studies |
Quality of life ‐ CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ Lumacaftor monotherapy vs placebo | no estimate extracted | 1 study · 85 participants | Low Downgraded: risk of bias, imprecision, indirectness |
FEV1 % predicted: relative change from baseline Summary of findings ‐ Lumacaftor monotherapy vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ Lumacaftor monotherapy vs placebo | no estimate extracted | 1 study · 61 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Adverse events Summary of findings ‐ Lumacaftor monotherapy vs placebo | no estimate extracted | 2 studies · 115 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ Lumacaftor monotherapy vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ Cavosonstat compared with placebo for cystic fibrosis
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ Cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Low Downgraded: risk of bias, imprecision, indirectness |
Quality of life: total score Summary of findings ‐ Cavosonstat vs placebo | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ Cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
FEV1 % predicted: relative change from baseline Summary of findings ‐ Cavosonstat vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ Cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Low Downgraded: risk of bias, imprecision, indirectness |
Adverse events: occurring in at least 10% of cavosonstat treated participants Summary of findings ‐ Cavosonstat vs placebo | no estimate extracted | 1 study · 26 participants | Very low Downgraded: risk of bias, imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ Cavosonstat vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ Lumacaftor plus ivacaftor (once daily) compared with placebo for cystic fibrosis (short term)
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ Lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1108 participants | High |
Quality of life ‐ (EuroQol) EQ‐5D‐3L Index Score (total score): absolute change from baseline Summary of findings ‐ Lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1061 participants | High |
Quality of life ‐ CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ Lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1076 participants | Moderate Downgraded: risk of bias |
FEV1 % predicted: relative change from baseline Summary of findings ‐ Lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1072 participants | High |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ Lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1072 participants | Moderate Downgraded: risk of bias |
Adverse events Summary of findings ‐ Lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1108 participants | High |
Time to first pulmonary exacerbation Summary of findings ‐ Lumacaftor plus ivacaftor (once daily) vs placebo | no estimate extracted | 2 studies · 1108 participants | Moderate Downgraded: risk of bias |
Summary of findings ‐ Lumacaftor plus ivacaftor (twice daily) compared with placebo for cystic fibrosis (short term)
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ Lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Quality of life ‐ total score Summary of findings ‐ Lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | NR | No studies |
Quality of life ‐ CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ Lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Low Downgraded: risk of bias, imprecision, indirectness |
FEV1 % predicted: relative change from baseline Summary of findings ‐ Lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ Lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Low Downgraded: risk of bias, imprecision, indirectness |
Adverse events Summary of findings ‐ Lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | 1 study · 204 participants | Low Downgraded: risk of bias, imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ Lumacaftor plus ivacaftor (twice daily) vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ Lumacaftor plus ivacaftor compared with placebo for cystic fibrosis (immediate term)
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ Lumacaftor plus ivacaftor vs placebo | no estimate extracted | 1 study · 62 participants | Moderate Downgraded: imprecision, indirectness |
Quality of life: total score Summary of findings ‐ Lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
Quality of life: respiratory domain Summary of findings ‐ Lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: relative change from baseline Summary of findings ‐ Lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ Lumacaftor plus ivacaftor vs placebo | no estimate extracted | 1 study · 59 participants | Moderate Downgraded: imprecision, indirectness |
Adverse events Summary of findings ‐ Lumacaftor plus ivacaftor vs placebo | no estimate extracted | 1 study · 61 participants | Low Downgraded: imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ Lumacaftor plus ivacaftor vs placebo | no estimate extracted | NR | No studies |
Summary of findings ‐ tezacaftor plus ivacaftor compared with placebo or ivacaftor alone
Population: adults and children with cystic fibrosis
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Survival Summary of findings ‐ tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 2 studies · 522 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Quality of life: total score Summary of findings ‐ tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | NR | No studies |
Quality of life: CFQ‐R respiratory domain: absolute change from baseline Summary of findings ‐ tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 2 studies · 522 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
FEV1 % predicted: relative change from baseline Summary of findings ‐ tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 2 studies · 522 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
FEV1 % predicted: absolute change from baseline Summary of findings ‐ tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 2 studies · 522 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Adverse events: most commonly occurring events (occurring in at least 10% of participants) Summary of findings ‐ tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 2 studies · 527 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |
Time to first pulmonary exacerbation Summary of findings ‐ tezacaftor plus ivacaftor vs placebo or ivacaftor alone | no estimate extracted | 1 study · 504 participants | Moderate Downgraded: risk of bias, imprecision, indirectness |