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血友病治疗的新方法:成功与挑战

Novel approaches to hemophilia therapy: successes and challenges.

作者信息

Arruda Valder R, Doshi Bhavya S, Samelson-Jones Benjamin J

机构信息

The Children's Hospital of Philadelphia, Philadelphia, PA.

Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA; and.

出版信息

Blood. 2017 Nov 23;130(21):2251-2256. doi: 10.1182/blood-2017-08-742312. Epub 2017 Oct 10.

Abstract

New therapies for hemophilia A and hemophilia B will likely continue to change clinical practice. Ranging from extended half-life to nonfactor products and gene therapy, these innovative approaches have the potential to enhance the standard of care by decreasing infusion frequency to increase compliance, promoting prophylaxis, offering alternatives to inhibitor patients, and easing route of administration. Each category has intrinsic challenges that may limit the broader application of these promising therapies. To date, none specifically address the challenge of dispersing treatment to the developing world.

摘要

血友病A和血友病B的新疗法可能会继续改变临床实践。从延长半衰期到非因子产品以及基因疗法,这些创新方法有潜力通过降低输注频率以提高依从性、促进预防、为抑制物患者提供替代方案以及简化给药途径来提升护理标准。每个类别都有其内在挑战,可能会限制这些前景广阔的疗法的更广泛应用。迄今为止,尚无一种疗法专门应对向发展中世界推广治疗的挑战。

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本文引用的文献

1
Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy.RNAi 治疗靶向血友病 A 或 B 的抗凝血酶。
N Engl J Med. 2017 Aug 31;377(9):819-828. doi: 10.1056/NEJMoa1616569. Epub 2017 Jul 10.
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Emicizumab Prophylaxis in Hemophilia A with Inhibitors.依库珠单抗预防伴抑制物的血友病 A。
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Targeting activated protein C to treat hemophilia.靶向活化蛋白C治疗血友病。
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SIPPET: methodology, analysis and generalizability.SIPPET:方法、分析与普遍性。
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