Hemophilia: is arevolution in treatment options taking place?

Abstract

Prophylactic replacement therapy for hemophiliaA (hereditary factor VIII deficiency) is asuccess story of the production of coagulation factor concentrates from donor plasma. Recombinant factor concentrates, which are also produced with modified gene constructs for coagulation factor VIII in order to improve pharmacological properties, have since proven their worth. This successful development over many years of factor concentrates for the successful treatment of hemophilia patients has now been followed by the innovation of afactor VIII mimetic in the form of amonoclonal antibody, which was developed in Japan already some years back. Emicizumab is ahumanized, bispecific monoclonal antibody for therapeutic use in hemophiliaA. With this therapeutic agent, the treatment of the hereditary coagulation defect is based, for the first time, on acompletely new active principle. The specific antibody simulates the properties of coagulation factor VIII as acofactor for the formation of the tenase complex with the coagulation factors IX and X. As a result under steady state conditions almost normal thrombin and thus fibrin formation can be achieved.