Standard treatments for patients with severe hemophilia A can be difficult, especially for children. They can also lead to the development of anti-factor VIII alloantibodies. In this study, researchers assessed emicizumab, which mimics the cofactor function of factor VIII, in 18 patients with severe hemophilia A.
Standard treatments for patients with severe hemophilia A can be difficult, especially for children. They can also lead to the development of anti-factor VIII alloantibodies. In this study, researchers assessed emicizumab, which mimics the cofactor function of factor VIII, in 18 patients with severe hemophilia A. For 12 weeks, patients received emicizumab at a weekly dose of 0.3, 1.0, or 3.0 mg/kg bodyweight. The treatment revealed no serious adverse effects or coagulation abnormalities. An exploratory endpoint, bleeding rate, was also analyzed. All three dosage cohorts experienced a decrease in bleeding rate (32.5 to 4.4, 18.3 to 0.0, and 15.2 to 0.0, respectively). Only 3 of 11 patients with factor VIII inhibitors experienced bleeding, as did 2 of 7 patients without factor VIII inhibitors had bleeding. The researchers concluded that the treatment was both safe and effective, reducing bleeding rates for hemophilia A patients with and without factor VIII inhibitors.
http://www.nejm.org/doi/full/10.1056/NEJMoa1511769
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