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1
Emicizumab-mediated haemostatic function in patients with haemophilia A is down-regulated by activated protein C through inactivation of activated factor V.依替巴肽介导的血友病 A 患者止血功能通过激活蛋白 C 使激活的因子 V 失活而受到下调。
Br J Haematol. 2018 Oct;183(2):257-266. doi: 10.1111/bjh.15525. Epub 2018 Aug 20.
2
Long-term outcomes from prophylactic or episodic treatment of haemophilia A: A systematic review.预防性或偶发性治疗血友病 A 的长期结局：系统评价。
Haemophilia. 2018 Sep;24(5):e301-e311. doi: 10.1111/hae.13546. Epub 2018 Jul 13.
3
Use of thrombin generation assay to personalize treatment of breakthrough bleeds in a patient with hemophilia and inhibitors receiving prophylaxis with emicizumab.使用凝血酶生成试验对一名患有血友病且产生抑制剂并接受艾美赛珠单抗预防治疗的患者的突破性出血进行个性化治疗。
Haematologica. 2018 Apr;103(4):e181-e183. doi: 10.3324/haematol.2017.185330. Epub 2018 Feb 22.
4
Long-term safety and efficacy of emicizumab in a phase 1/2 study in patients with hemophilia A with or without inhibitors.艾美赛珠单抗在伴或不伴抑制剂的A型血友病患者1/2期研究中的长期安全性和疗效
Blood Adv. 2017 Sep 27;1(22):1891-1899. doi: 10.1182/bloodadvances.2017006684. eCollection 2017 Oct 10.
5
A Pharmacometric Approach to Substitute for a Conventional Dose-Finding Study in Rare Diseases: Example of Phase III Dose Selection for Emicizumab in Hemophilia A.一种替代罕见疾病常规剂量研究的药物计量学方法：以血友病 A 中emicizumab 的 III 期剂量选择为例。
Clin Pharmacokinet. 2018 Sep;57(9):1123-1134. doi: 10.1007/s40262-017-0616-3.
6
Emicizumab, a bispecific antibody recognizing coagulation factors IX and X: how does it actually compare to factor VIII?依库珠单抗，一种识别凝血因子 IX 和 X 的双特异性抗体：它与因子 VIII 相比实际情况如何？
Blood. 2017 Dec 7;130(23):2463-2468. doi: 10.1182/blood-2017-08-801662. Epub 2017 Oct 17.
7
Emicizumab Prophylaxis in Hemophilia A with Inhibitors.依库珠单抗预防伴抑制物的血友病 A。
N Engl J Med. 2017 Aug 31;377(9):809-818. doi: 10.1056/NEJMoa1703068. Epub 2017 Jul 10.
8
Factor VIIIa-mimetic cofactor activity of a bispecific antibody to factors IX/IXa and X/Xa, emicizumab, depends on its ability to bridge the antigens.双特异性抗体emicizumab对因子IX/IXa和X/Xa的VIIIa模拟辅因子活性取决于其桥接抗原的能力。
Thromb Haemost. 2017 Jun 28;117(7):1348-1357. doi: 10.1160/TH17-01-0030. Epub 2017 Apr 28.
9
Prophylaxis usage, bleeding rates, and joint outcomes of hemophilia, 1999 to 2010: a surveillance project.1999年至2010年血友病的预防用药、出血率及关节转归：一项监测项目
Blood. 2017 Apr 27;129(17):2368-2374. doi: 10.1182/blood-2016-02-683169. Epub 2017 Feb 9.
10
A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A.A 随机对照试验VIII 因子和中和抗体在血友病 A.
N Engl J Med. 2016 May 26;374(21):2054-64. doi: 10.1056/NEJMoa1516437.

依库珠单抗的使用不应依赖于免疫耐受诱导。

Emicizumab should be prescribed independent of immune tolerance induction.

机构信息

Clinical Hemostasis Unit, Department of Hematology, Louis Pradel University Hospital, Bron, France; and.

EA 4609 Hemostasis and Cancer, University Claude Bernard Lyon I, Lyon, France.

出版信息

Blood Adv. 2018 Oct 23;2(20):2783-2786. doi: 10.1182/bloodadvances.2018015859.

DOI:10.1182/bloodadvances.2018015859
PMID:30352952
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6199655/
Abstract

This article has a companion Point by Young.

摘要

本文有 Young 的相关观点。