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Evidence found and GRADE certainty

11 questions · 11 findings · 2 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 0 · 0%GRADE not reported 1 · 9.1%Very low 2 · 18.2%Low 2 · 18.2%Moderate 6 · 54.5%High 0 · 0%

Long‐term red cell transfusion vs hydroxyurea and phlebotomy

Serious adverse events as a result of sickle cell‐related complications · individuals with sickle cell disease who are at risk of a primary stroke who have had long‐term red cell transfusions to prevent a stroke

It is very uncertain whether long‐term red cell transfusion changes serious adverse events as a result of sickle cell‐related… compared with hydroxyurea and phlebotomy (very low certainty).

1 finding from 1 review

Long‐term red cell transfusion vs hydroxyurea and phlebotomy

Serious adverse events as a result of sickle cell‐related complications · individuals with sickle cell disease who have had a stroke who have had long‐term red cell transfusions to prevent another stroke

It is very uncertain whether long‐term red cell transfusion changes serious adverse events as a result of sickle cell‐related… compared with hydroxyurea and phlebotomy (very low certainty).

1 finding from 1 review

Long‐term red cell transfusion vs no transfusion

Serious adverse events as a result of sickle cell‐related complications · individuals with sickle cell disease who are at risk of a primary stroke who have not had previous long‐term red cell transfusions

Long‐term red cell transfusion may leads to lower serious adverse events as a result of sickle cell‐related… compared with no transfusion (low certainty).

1 finding from 1 review

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

Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy

Serious adverse events · people without inhibitors

Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis showed no clear difference in serious adverse events 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

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 ‐ 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 ‐ 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