Nonfactor Therapies for Hemophilia: Current Evidence, Clinical Relevance and Cost Considerations | Managed Healthcare Executive

Nonfactor Therapies for Hemophilia

Hemophilia is a congenital, X-linked bleeding disorder caused by deficiencies in coagulation factor VIII (FVIII) or factor IX (FIX) due to mutations in the F8 or F9 genes on the X chromosome.

These deficiencies impair thrombin generation, resulting in a lifelong predisposition to bleeding. Hemophilia A is more common, with a prevalence of approximately 1 in 4,000 male live births, compared to 1 in 20,000 male live births for hemophilia B.

Female carriers are about 1.6 times more prevalent than affected males, with some carriers experiencing reduced factor levels and clinically significant bleeding.

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Author's summary: Hemophilia is a genetic bleeding disorder with two main types, A and B.

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Managed Healthcare Executive Managed Healthcare Executive — 2025-10-31

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