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血友病重组因子替代疗法的演变

The evolution of recombinant factor replacement for hemophilia.

作者信息

Sankar Amanda D, Weyand Angela C, Pipe Steven W

机构信息

Department of Pediatrics, University of Michigan, Ann Arbor, MI, USA.

Department of Pediatrics, University of Michigan, Ann Arbor, MI, USA.

出版信息

Transfus Apher Sci. 2019 Oct;58(5):596-600. doi: 10.1016/j.transci.2019.08.010. Epub 2019 Aug 9.

Abstract

Hemophilia A and Hemophilia B are the most common of the severe bleeding disorders and are caused by a deficiency in blood clotting factor VIII or factor IX respectively. Factor replacement therapy has been the cornerstone of treatment to treat life threatening bleeds and prevent joint disease. The treatment of hemophilia has evolved tremendously over the past five decades from fresh frozen plasma as the only available therapy to more specific plasma-derived and recombinant-derived factor replacement. Now due to innovations in bioengineering, there are even more efficacious factor replacement options available to patients. Here we review these recent advancements and their impact on the treatment and management of hemophilia.

摘要

甲型血友病和乙型血友病是最常见的严重出血性疾病,分别由凝血因子VIII或因子IX缺乏引起。因子替代疗法一直是治疗危及生命的出血和预防关节疾病的治疗基石。在过去的五十年里,血友病的治疗取得了巨大进展,从仅有的新鲜冷冻血浆疗法发展到更具特异性的血浆源性和重组源性因子替代疗法。如今,由于生物工程领域的创新,患者有了更多有效的因子替代选择。在此,我们回顾这些最新进展及其对血友病治疗和管理的影响。

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