The current and future role of plasma-derived clotting factor concentrate in the treatment of haemophilia A

Abstract

While recent advances in recombinant DNA technology, gene therapy and the discovery of novel non-substitutional agents have revolutionised the management of haemophilia A, plasma-derived clotting factor concentrates continue to be used and are likely to remain an important therapeutic option for the foreseeable future. The administration of plasma-derived products is associated with a risk of pathogen transmission. However, this risk has been greatly reduced, if not virtually eliminated, by modern manufacturing techniques. Moreover, FVIII inhibitor formation (which has overtaken pathogen transmission as the major modern complication of factor replacement therapy) may occur less frequently with the use of plasma-derived FVIII concentrates relative to recombinant alternatives, although this remains a source of considerable debate. In addition, plasma-derived FVIII concentrates remain the primary replacement therapy for haemophilia A in the developing world, where access to recombinant therapy is more limited. The future role of plasma-derived concentrates globally will be determined by several factors including the ability of manufacturers to continue to provide safe and affordable product, the potential for plasma-derived concentrates to confer lower a risk of inhibitor formation in previously untreated patients with haemophilia and the impact of novel therapies on the market for plasma-derived products both in the developed and developing world.