Another Victory for Patients with Hemophilia

Congenital hemophilia A is a rare bleeding disorder caused by a mutation in the gene encoding factor VIII, resulting in a deficiency of factor VIII activity. Severe hemophilia (factor VIII activity level, <1%) is characterized by repetitive bleeding into joints beginning in early childhood and poses a major risk of life-threatening hemorrhage. Moderate hemophilia (factor VIII activity level, 1 to 5%) is associated with less frequent joint and soft-tissue bleeding related to mild trauma. Replacement therapy with the use of factor VIII concentrates restores hemostasis by raising levels of factor VIII activity and is effective.