The hemophilias and their clinical management

摘要

Outcomes for patients with hemophilia have improved dramatically over the past 50 years. With the increased availability of safe clotting factor concentrates, the primary focus in clinical management is now the prevention of long-term complications, most notably the debilitating hemophilic arthropathy that is associated with severe disease. This article reviews evidence-based approaches for managing both children and adults with hemophilia. Definitive evidence of improved clinical results from primary prophylaxis started in young patients with severe hemophilia A and a minimal bleeding history is presented. Furthermore, recent studies showing benefits for initiating prophylaxis in older adolescents and adults with established joint disease are examined. Inhibitors to factor VIII are the most problematic complication of factor replacement therapy. Patient-specific and treatment-related factors that contribute to the risk of inhibitor formation are discussed and controversies and clinical evidence related to approaches for tolerance induction are reviewed.