title: Trends in novel treatments for hemophilia.
abstract: The principle of hemophilia treatment is replacement therapy with factor VIII and factor IX concentrates. Recently, extended half-life factor VIII and factor IX concentrates have been developed. With these concentrates, improvements in patient QOL can be expected. More recently, a novel hemophilia therapy based on a very new concept was developed. ACE910 (emicizumab) is a humanized bispecific antibody recognizing factor IXa and X mimicking factor VIII function. The half-life is reportedly 4-5 weeks and remarkably decreased annual bleeding rates have been achieved with subcutaneous weekly injections in the phase 1 clinical trial. Clinical trials of anti-antithrombin therapeutics based on siRNA (ALN-AT3) and anti-TFPI antibody are currently ongoing and the results are eagerly anticipated.
authors: Department of Pediatrics, Nara Medical University.
authors: Midori
authors: M
authors: Shima
mesh_terms: D018033:Antibodies, Bispecific / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D005343:Fibrinolytic Agents / Q000627:therapeutic use; D006467:Hemophilia A / Q000276:immunology / Q000628:therapy*; D006801:Humans; D034741:RNA, Small Interfering / Q000235:genetics / Q000627:therapeutic use
chemical_list: D018033:Antibodies, Bispecific; D005343:Fibrinolytic Agents; D034741:RNA, Small Interfering