Findings

What do systematic reviews say?

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Evidence found and GRADE certainty

54 questions · 54 findings · 2 reviews · Cystic Fibrosis and Genetic Disorders

No studies found 2 · 3.7%GRADE not reported 0 · 0%Very low 2 · 3.7%Low 14 · 25.9%Moderate 36 · 66.7%High 0 · 0%

Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy

Proportion with zero bleeds · people with inhibitors

Summary of findings table ‐ Fitusiran prophylaxis probably leads to higher proportion with zero bleeds compared with on‐demand therapy (moderate certainty).

1 finding from 1 review

Summary of findings table ‐ Concizumab prophylaxis vs on‐demand therapy

Adverse events ‐ All adverse events · people with inhibitors

Summary of findings table ‐ Concizumab prophylaxis showed no clear difference in adverse events ‐ All adverse events compared with on‐demand therapy (low certainty).

1 finding from 1 review

Summary of findings table ‐ Concizumab prophylaxis vs on‐demand therapy

Adverse events ‐ Serious adverse events · people with inhibitors

Summary of findings table ‐ Concizumab prophylaxis showed no clear difference in adverse events ‐ Serious adverse events compared with on‐demand therapy (low certainty).

1 finding from 1 review

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

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