Findings

What do systematic reviews say?

7 questions · 7 findings · 1 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 0 · 0%GRADE not reported 0 · 0%Very low 0 · 0%Low 1 · 14.3%Moderate 6 · 85.7%High 0 · 0%

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

Annualized Bleeding Rate (ABR) ‐ All treated bleeds · people with 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 · 53 participants
Moderate

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

Annualized spontaneous Bleeding Rate (AsBR) · people with 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 · 53 participants
Moderate

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

Annualized joint Bleeding Rate (AjBR) · people with 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 · 53 participants
Moderate

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

Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B 2024 · Cochrane Headline outcomes only
RR 11.31 [1.66, 77.3]
1 study · 53 participants
Moderate

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

QoL · people with 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 · 40 participants
Low

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

All adverse events · people with inhibitors

Summary of findings table ‐ Emicizumab 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 1.97 [1.26, 3.1]
1 study · 52 participants
Moderate

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

Serious adverse events · people with inhibitors

Summary of findings table ‐ Emicizumab 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 1.32 [0.48, 3.63]
1 study · 52 participants
Moderate