Findings

What do systematic reviews say?

873 questions · 883 findings · 38 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 319 · 36.1%GRADE not reported 18 · 2%Very low 198 · 22.4%Low 186 · 21.1%Moderate 147 · 16.6%High 15 · 1.7%

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 ‐ 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 table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy

Annualized Bleeding Rate (ABR) ‐ All treated bleeds · people without inhibitors

1 finding from 1 review

Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B 2024 · Cochrane Headline outcomes only
no estimate extracted
1 study · 54 participants
Moderate

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy

Annualized joint Bleeding Rate (AjBR) · people without inhibitors

1 finding from 1 review

Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B 2024 · Cochrane Headline outcomes only
no estimate extracted
1 study · 54 participants
Moderate

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy

Annualized spontaneous Bleeding Rate (AsBR) · people without inhibitors

1 finding from 1 review

Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B 2024 · Cochrane Headline outcomes only
no estimate extracted
1 study · 54 participants
Moderate

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy

Proportion with zero bleeds · people without inhibitors

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis may leads to higher proportion of participants with zero bleeds compared with on‐demand therapy (low certainty).

1 finding from 1 review

Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B 2024 · Cochrane Headline outcomes only
RR 19 [1.21, 298.4]
1 study · 54 participants
Low

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy

QoL · people without inhibitors

1 finding from 1 review

Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B 2024 · Cochrane Headline outcomes only
no estimate extracted
1 study · 47 participants
Low

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy

All adverse events · people without inhibitors

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis probably leads to higher all adverse events compared with on‐demand therapy (moderate certainty).

1 finding from 1 review

Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B 2024 · Cochrane Headline outcomes only
RR 2.83 [1.47, 5.47]
1 study · 54 participants
Moderate

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy

Serious adverse events · people without inhibitors

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis showed no clear difference in serious adverse events compared with on‐demand therapy (moderate certainty).

1 finding from 1 review

Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B 2024 · Cochrane Headline outcomes only
RR 0.5 [0.03, 7.54]
1 study · 54 participants
Moderate

Summary of findings: amikacin liposome inhalation suspension (ALIS) vs tobramycin

FEV1 · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 262 participants
Moderate