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Search review questions

Each row is one question a systematic review asked: who, what, compared with what, and which outcome, with the effect it found and how certain that is. Words match anywhere in the field.

Limit to evidence status or GRADE (optional)

2,280 questions in 114 reviews · Cystic Fibrosis and Genetic Disorders

current versions
No studies 856 · 37.5%Ungraded 58 · 2.5%Very low 550 · 24.1%Low 502 · 22%Moderate 271 · 11.9%High 43 · 1.9%

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.

QuestionEffectEvidenceStatus / GRADE
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo
Time to first pulmonary exacerbation · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo
Survival · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 62 participantsModerate
Summary of findings ‐ monotherapy: lumacaftor vs placebo
Quality of life ‐ CFQ‐R respiratory domain: absolute change from baseline · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 85 participantsLow
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo
Quality of life: total score · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo
Quality of life: respiratory domain · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo
FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo
FEV1 % predicted: absolute change from baseline · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 59 participantsModerate
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo
Adverse events · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 61 participantsLow
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo
Time to first pulmonary exacerbation · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone
Survival · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted9 studies · 1132 participantsLow
Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone
Quality of life: total score · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
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
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted5 studies · 946 participantsLow
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
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted5 studies · 944 participantsLow
Summary of findings ‐ monotherapy: lumacaftor vs placebo
FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
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
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted5 studies · 944 participantsLow
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
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted8 studies · 1057 participantsModerate
Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone
Time to first pulmonary exacerbation · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 506 participantsModerate
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
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted4 studies · 634 participantsModerate
Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control
Quality of life: total score · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control
Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 382 participantsModerate
Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control
FEV1 (% predicted): relative change from baseline · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted6 participantsUngraded
Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control
FEV1 (% predicted): absolute change from baseline · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 382 participantsModerate
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
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted4 studies · 634 participantsModerate
Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control
Time to first pulmonary exacerbation · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ monotherapy: lumacaftor vs placebo
FEV1 % predicted: absolute change from baseline · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 61 participantsModerate
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control
Survival · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted3 studies · 603 participantsHigh
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control
Quality of life: total score · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control
Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted3 studies · 599 participantsModerate
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control
FEV1 (% predicted): relative change from baseline · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted3 studies · 603 participantsModerate
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control
FEV1 (% predicted): absolute change from baseline · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted2 studies · 578 participantsModerate
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
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted3 studies · 603 participantsModerate
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control
Time to first pulmonary exacerbation · adults with cystic fibrosis and either F508del/MF or F508del/F508del genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)
Survival · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual…
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 258 participantsModerate
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)
Quality of life: total score · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual…
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)
Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual…
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ monotherapy: lumacaftor vs placebo
Adverse events · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted2 studies · 115 participantsVery low
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)
FEV1 (% predicted): relative change from baseline · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual…
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)
FEV1 (% predicted): absolute change from baseline · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual…
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 258 participantsModerate
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)
Adverse events: number of participants with treatment‐emergent adverse events · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual…
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
OR 1.04 [0.53, 2.04]1 study · 258 participantsModerate
Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)
Time to first pulmonary exacerbation · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual…
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo
Survival · adults with cystic fibrosis and with the F508del/MF genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 55 participantsLow
Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo
Quality of life: total score · adults with cystic fibrosis and with the F508del/MF genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo
Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults with cystic fibrosis and with the F508del/MF genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 55 participantsLow
Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo
FEV1 (% predicted): relative change from baseline · adults with cystic fibrosis and with the F508del/MF genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 55 participantsLow
Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo
FEV1 (% predicted): absolute change from baseline · adults with cystic fibrosis and with the F508del/MF genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 55 participantsLow
Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo
Adverse events · adults with cystic fibrosis and with the F508del/MF genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 55 participantsLow
Summary of findings ‐ monotherapy: lumacaftor vs placebo
Time to first pulmonary exacerbation · adults and children with cystic fibrosis
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo
Time to first pulmonary exacerbation · adults with cystic fibrosis and with the F508del/MF genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies
Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo
Survival · adults with cystic fibrosis and with the F508del/MF genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extracted1 study · 76 participantsLow
Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo
Quality of life: total score · adults with cystic fibrosis and with the F508del/MF genotype
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane
no estimate extractedNRNo studies