Factor concentrates for treatment of hemophilia: which one to choose?

U NTIL THE ADVENT of components that could more specifically replace specific missing clotting factors, patients with hemophilia were treated with whole blood and then plasma. Such patients had a shortened lifespan and were crippled with severe anthropathy; many died of hemonrhage. With the discovery in 1960 that factor VIII:C was concentrated in cryoprecipitate, more specific replacement for persons with factor FVIII deficiency was possible. In the early 1 970s, further purification processes were discovered, and lyophihized concentrates of higher purity, both factor VIII and pnothnombin complex concentrates (PCCs), began to be produced. Lyophilized concentrates revolutionized the care of patients with hemophilia. In home therapy programs, patients were taught to infuse the concentrate in the home setting at the earliest sign of hemorrhage.’ The long-term effects of hemorrhage were decreased, days lost from work or school and days of hospitalization decreased, and lifespan gradually increased until the AIDS era.2’3 Lyophilized concentrate of clotting factors are prepared from plasma obtained from 2,000 to 30,000 donors. Infectious complications from transfusion-transmitted viruses began to be noted in patients with hemophilia in the late 1970s and are still a major concern. Many newer types of factor concentrates are being produced, both to eliminate infectious side effects and increase purity. This review first describes the complications associated with concentrate infusion and then reviews the various factor concentrate products currently available for treatment of hemophilia and their advantages and disadvantages. The treatment of patients with inhibitors is beyond the scope of this review and is the subject of several recent articles.4’5