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- Midori Shima.
- Department of Pediatrics, Nara Medical University.
- Rinsho Ketsueki. 2016 Jan 1; 57 (10): 2259-2266.
AbstractThe 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.
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