Genetics and Factor Dose Influence Inhibitor Development in Hemophilia A  

A study published in Blood Advances found that the F8 genotype and higher factor dose are risk factors for developing inhibitors in patients with severe hemophilia A. The PUPs A-LONG study evaluated the safety and efficacy of efmoroctocog alfa in previously untreated patients with severe hemophilia A, showing an incidence of inhibitor development of approximately 31.1%. Further analyses revealed that certain F8 variants increased the risk of inhibitor development, while higher doses of treatment in patients with low-titer inhibitors could lead to progression into high-titer inhibitors. The study also suggested the need for exploration into the relationship between central venous access devices and hemophilia severity.

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