Categories: Hemophilia

November 2004 Volume 2, Issue 11

Christine L. Kempton, MD, Miguel A. Escobar, MD, and Harold R. Roberts, MD

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Hemophilia A and hemophilia B are X-linked disorders caused by defi cient or defective factor VIII or factor IX molecules, respectively. The care of patients with hemophilia has improved dramatically beginning with the ability to purify factor concentrates. These improvements have led to a significant improvement in the quality of life enjoyed by hemophiliacs. More recent advances in hemophilia care have included the development of new recombinant factor VIII and factor IX products with reduced or absent added human or animal proteins, preclinical development of factor VIII molecules with altered and more favorable pharmacokinetics and immunogenicity, and the development of economically feasible methods to produce plasma-derived products free of transmissible agents for use in developing countries. Gene therapy continues to hold promise for patients with severe disease. Other longstanding issues which require continuing research include: improvement in methods to prevent or treat patients with inhibitors such as immune tolerance and immune modulation; the establishment of optimal dosing of recombinant factor VIIa (rfVIIa); and further understanding of the possible synergy between rfVIIa agents that appear to improve the effi cacy of rfVIIa such as FEIBA, prothrombin, and factor X.