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止血治疗作为获得性血友病的一种管理策略:未来会怎样?

Hemostatic therapy as a management strategy for acquired hemophilia: what does the future hold?

作者信息

Franchini Massimo, Schiavulli Michele, Liumbruno Giancarlo Maria

机构信息

Department of Hematology and Transfusion Medicine, Carlo Poma Hospital, Mantova, Italy.

Regional Reference Center for Coagulation Disorders, Santobono-Pausilipon Hospital, Naples, Italy.

出版信息

Expert Rev Hematol. 2021 Mar;14(3):263-270. doi: 10.1080/17474086.2021.1892483. Epub 2021 Mar 3.

Abstract

: Acquired hemophilia A (AHA) is a rare autoimmune disease caused by autoantibodies that bind and inactivate factor VIII (FVIII), predisposing to a potentially life-threatening bleeding.: The main epidemiological, clinical, laboratory and therapeutic features of AHA are critically discussed. In particular, we focus on the hemostatic management of AHA patients analyzing the currently available treatment options and showing the latest data on the innovative hemostatic agents still under investigation. Authors searched the Medline and PubMed electronic databases for publication on AHA in the last twenty years.: While a rapid recognition of suspected cases of AHA is essential to make a correct diagnosis and appropriately and timely treat the hemorrhagic manifestations, the multidisciplinary approach to this challenging, rare and life-threatening bleeding disorder is equal importance to improve patients' outcome. Although promising, the safety and efficacy of the clinical use of emicizumab in AHA needs to be validated by trials including an adequate number of patients, before registering the drug also for this indication.

摘要

获得性血友病A(AHA)是一种罕见的自身免疫性疾病,由自身抗体引起,该抗体可结合并使凝血因子VIII(FVIII)失活,从而易引发危及生命的出血。本文对AHA的主要流行病学、临床、实验室及治疗特征进行了批判性讨论。特别地,我们聚焦于AHA患者的止血管理,分析了当前可用的治疗选择,并展示了仍在研究中的创新止血药物的最新数据。作者检索了Medline和PubMed电子数据库,以获取过去二十年中关于AHA的出版物。虽然快速识别AHA疑似病例对于正确诊断以及适当及时治疗出血表现至关重要,但对于这种具有挑战性、罕见且危及生命的出血性疾病,多学科方法对于改善患者预后同样重要。虽然艾美赛珠单抗在AHA临床应用中前景可期,但其安全性和有效性在该药物获批此适应症之前,仍需通过纳入足够数量患者的试验来验证。

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