Choice of replacement therapy for hemophilia: recombinant products only?

Abstract

In the early 1980s it was recognized that coagulation factor concentrates prepared from plasma pooled from thousands of donors transmitted the hepatitis and the human immunodeficiency viruses. The last 15 years have witnessed the development of methods that, applied during concentrate manufacturing, inactivate viruses escaping the screening procedures. The adoption of these measures has dramatically reduced the risk of transmission of bloodborne infections by plasma-derived coagulation factor concentrates. The production of recombinant factor VIII and IX and their availability for patient treatment epitomize progress in hemophilia care through DNA technology. No transmission of infectious agents has been documented and the small amount of residual animal proteins, particularly in the early products, has not been associated with any clinically significant adverse reaction. There is little doubt that recombinant factor VIII and IX are the products of choice for the treatment of patients with hemophilia, particularly the second-generation products containing no plasma-derived human protein and fewer and fewer animal proteins. However, availability and cost still oblige most physicians to ration recombinant products and prioritize certain patient groups (previously untreated, previously uninfected).