Treatment of Haemophilia A and B and von Willebrand's Disease

Abstract

This review aims to assess – from medical, economic, and ethical perspectives – different dosing strategies for replacement therapy using coagulation factor concentrates to treat patients with hemophilia A and B and von Willebrand disease.The systematic literature review does not cover the safety aspects related to transmission of infections, or the risks for developing inhibitors (neutralising antibodies). The methods currently used for virus inactivation and virus reduction in producing plasma-based factor concentrates have been approved by European and U.S. drug authorities, and the productsare considered to have a wide margin of safety. The transition to products manufactured by recombinant DNA methods has favourably altered the risks of blood contamination. An international debate is under way concerning the risks of inhibitor development, but evidence supporting the various opinions is deficient. Even a minor difference could have considerable medical and economic consequences.