Current status and future perspective of treatment for hemophilia

Abstract

Hemophiliacs are X-linked inherited bleeding disorders. The treatment of hemophilia is based on periodic replacement treatment with coagulation factor products to avoid the development of hemophilic arthropathy, as well as to control fatal bleeding. However, because of the coagulation factor's incredibly short half-life, the medication must be often given intravenously throughout one's life. Extended half-life coagulation factor preparations have lowered the burden of treatment by enhancing pharmacokinetics. However, the patients remain at risk of developing inhibitors to significantly interfere with the therapeutic effectiveness of coagulation factor concentrates. Recently, nonfactor replacement therapies have gained a lot of interest as a new class of therapeutic drugs preventing bleeding in patients with or without inhibitors. Moreover, gene therapy drugs for hemophilia have recently been approved in Europe and the United States. Because gene therapy can preserve coagulation factor levels in the blood for at least several years with a single treatment, it may become a curative treatment that removes the need for the therapy for a long time.