Recent advances in the development of coagulation factors and procoagulants for the treatment of hemophilia.

摘要: Hemophilia is a family of rare bleeding disorders. The two primary types, hemophilia A and B, are caused by recessive X-chromosome linked mutations that result in deficiency coagulation factor VIII (FVIII) or IX (FIX), respectively. Clinically, manifested spontaneous bleeding, particularly into the joints (haemarthrosis) soft tissue, excessive following trauma surgery. total overall number patients worldwide approximately 400,000, however only about 100,000 these individuals treated. first treatment was initiated when it determined clotting could be corrected plasma fraction taken from normal blood. discovery enrichment cryoprecipitation opened new era therapy which eventually led to production concentrates subsequent development highly purified forms factors. most significant improvements have been availability recombinant factors IX. Unfortunately, still retain some limitations concentrates. These include antibody responses relatively short half-life molecules requiring frequent injection maintain effective concentration. Treatment beyond replacement native has tried. They modified with improved potency, stability circulating enhancement prothrombotic and/or stabilization via inhibition key negative regulatory pathways. approaches will reviewed this commentary.