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临床复杂型血友病 A 患者抑制物消除的重要性。

The importance of inhibitor eradication in clinically complicated hemophilia A patients.

机构信息

a Institute of Experimental Hematology and Transfusion Medicine , University Clinic Bonn , Bonn , Germany.

b Hemostasis and Thrombosis Center Children's Hospital , Los Angeles , CA , USA.

出版信息

Expert Rev Hematol. 2018 Nov;11(11):857-862. doi: 10.1080/17474086.2018.1521718. Epub 2018 Oct 4.

Abstract

Inhibitors against factor VIII (FVIII), which develop in around 20-30% of patients with severe hemophilia A, represent a significant complication of on-demand or prophylactic FVIII therapy. Currently, the main treatment option for inhibitor patients is eradication using immune tolerance induction (ITI) therapy. Areas covered: This article reviews inhibitor eradication in clinically complicated hemophilia A patients. The benefits and disadvantages of ITI therapy are discussed, with reference to the new nonfactor replacement agents such as emicizumab, which are becoming available. Expert commentary: At present, ITI therapy is the method of choice for all inhibitor patients, but as alternative treatment options become increasingly available, this may change in the future. Despite the development of nonfactor replacement agents, ITI therapy is likely to maintain a key role in the management of inhibitor patients, although changes in ITI therapy regimens including the timing of ITI initiation, the dosing regimen, the management of thrombotic risk or safety, laboratory assessment of ITI outcomes, and maintenance treatment after ITI success, may be necessary.

摘要

针对因子 VIII(FVIII)的抑制剂会在大约 20-30%的重型 A 型血友病患者中产生,这是按需或预防性 FVIII 治疗的一个重大并发症。目前,抑制剂患者的主要治疗选择是使用免疫耐受诱导(ITI)疗法进行清除。

涵盖领域

本文回顾了临床上复杂的 A 型血友病患者的抑制剂清除。讨论了 ITI 治疗的优缺点,并参考了emicizumab 等新型非因子替代物,这些药物正逐渐应用于临床。

专家评论

目前,ITI 疗法是所有抑制剂患者的首选方法,但随着替代治疗选择的日益增多,这种情况在未来可能会发生变化。尽管出现了非因子替代物,但 ITI 疗法可能仍将在抑制剂患者的管理中发挥关键作用,尽管 ITI 治疗方案可能需要改变,包括 ITI 起始时间、剂量方案、血栓风险或安全性管理、ITI 结果的实验室评估以及 ITI 成功后的维持治疗等方面。

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