Blood products for hemophilia: past, present and future.

Abstract

Hemophilia is an inherited bleeding disorder, which in its severe form is characterized by recurrent hemarthrosis and internal bleeding. In the absence of effective treatment the prognosis is poor, but the development of blood products in the last few decades has transformed the outlook, and patients can now live essentially normal lives. Treatment options vary around the world, with cryoprecipitate still the mainstay of therapy in many developing countries. Many patients were infected with hepatitis and/or HIV through the use of coagulation factor concentrates before the introduction of physical methods of viral inactivation in the mid-1980s. In more affluent countries, the debate in recent years has focused on the relative merits of plasma versus recombinant products. Coagulation factor concentrates are expensive, and cost-benefit and quality-of-life studies will assume an increasing importance in guiding the selection of products. Looking to the future, genetic engineering offers the potential to create coagulation factors with enhanced properties, such as reduced immunogenicity and prolonged half-life. Transgenic animals are a potential source of therapeutic materials. Several trials of gene therapy for hemophilia are already underway.