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Evidence found and GRADE certainty

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 table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks

Proportion with zero bleeds · people without inhibitors

Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly showed no clear difference in proportion of participants with zero bleeds compared with emicizumab 3.0 mg/kg every 2 weeks (moderate certainty).

1 finding from 1 review

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

Proportion with zero bleeds · people with inhibitors

Summary of findings table ‐ Emicizumab 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 ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks

All adverse events · people without inhibitors

Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly showed no clear difference in all adverse events compared with emicizumab 3.0 mg/kg every 2 weeks (moderate certainty).

1 finding from 1 review

Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks

Serious adverse events · people without inhibitors

Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly showed no clear difference in serious adverse events compared with emicizumab 3.0 mg/kg every 2 weeks (moderate certainty).

1 finding from 1 review

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

Proportion with zero bleeds · people without 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 ‐ Fitusiran prophylaxis vs on‐demand therapy

Adverse events ‐ Serious adverse events · people without inhibitors

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

1 finding from 1 review