Findings

What do systematic reviews say?

2,261 questions · 2,280 findings · 114 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 856 · 37.5%GRADE not reported 58 · 2.5%Very low 550 · 24.1%Low 502 · 22%Moderate 271 · 11.9%High 43 · 1.9%

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

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 ‐ 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

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 · 55 participants
Low

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

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 · 55 participants
Low

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

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 · 55 participants
Low

Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo

Adverse events · adults with cystic fibrosis and with the F508del/MF 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
1 study · 55 participants
Low

Summary of findings ‐ monotherapy: lumacaftor 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 ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo

Time to first pulmonary exacerbation · adults with cystic fibrosis and with the F508del/MF genotype

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 ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo

Survival · adults with cystic fibrosis and with the F508del/MF 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
1 study · 76 participants
Low

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

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 ‐ triple therapy: VX‐152‐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

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 · 76 participants
Low

Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults with cystic fibrosis and with the F508del/MF 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
1 study · 76 participants
Low

Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo

FEV1 % predicted: absolute change from baseline · adults with cystic fibrosis and with the F508del/MF 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
1 study · 76 participants
Low

Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo

Adverse events · adults with cystic fibrosis and with the F508del/MF 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
1 study · 76 participants
Low

Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo

Time to first pulmonary exacerbation · adults with cystic fibrosis and with the F508del/MF genotype

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 ‐ monotherapy: cavosonstat 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 · 26 participants
Very low

Summary of findings ‐ monotherapy: cavosonstat 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

Intervention not reported

50% reduction of tumour sizea · people with TSC

The intervention leads to higher 50% reduction of tumour sizea (high certainty).

1 finding from 1 review

Rapamycin and rapalogs for tuberous sclerosis complex 2023 · Cochrane Headline outcomes only
RR 24.7 [3.5, 173.4]
2 studies · 162 participants
High

Intervention not reported

Frequency of seizure · people with TSC

The review found no eligible studies for this question.

1 finding from 1 review

Rapamycin and rapalogs for tuberous sclerosis complex 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Intervention not reported

Creatinine level · people with TSC

The review found no eligible studies for this question.

1 finding from 1 review

Rapamycin and rapalogs for tuberous sclerosis complex 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Intervention not reported

FEV1/FVC ratio · people with TSC

The review found no eligible studies for this question.

1 finding from 1 review

Rapamycin and rapalogs for tuberous sclerosis complex 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found