Acquired hemophilia A: a narrative review and management approach in the emicizumab era.

IF 5.5 2区 医学 Q1 HEMATOLOGY
Patrick Ellsworth, Sheh-Li Chen, Lee Ann Jones, Alice D Ma, Nigel S Key
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引用次数: 0

Abstract

Acquired hemophilia A (AHA) is a rare bleeding disorder caused by inhibitory autoantibodies to factor (F)VIII. The goals of treatment are 2-fold, namely immunosuppressive therapy to eradicate the inhibitor and hemostatic management to control bleeding. Emicizumab, a bispecific antibody that acts as a FVIIIa-mimetic, has seen growing use in AHA following its approval for congenital hemophilia A. This review provides an overview of the epidemiology, pathophysiology, diagnosis, and treatment of AHA. Registry, trial, and case series data are assimilated and summarized with an emphasis on a standardized approach that integrates the use of emicizumab. With recent registry data suggesting the need to focus on immunosuppression-related mortality in AHA, we provide treatment recommendations in an algorithmic format that have become the standard of care at our institution. These recommendations are intended to minimize hemostatic product usage and potential toxicity related to immunosuppressive therapy while reducing morbidity and rehospitalization rates for bleeding. The proposed treatment algorithm, which includes key interventions by phase of therapy, can be readily implemented at centers that have rapid access to plasma FVIII activity using a one-stage assay. A case is presented to illustrate the proposed diagnostic and management considerations.

获得性血友病 A:Emicizumab 时代的叙事回顾和管理方法。
获得性血友病 A(AHA)是一种罕见的出血性疾病,由因子 VIII(FVIII)抑制性自身抗体引起。治疗的目标有两个方面,一是通过免疫抑制疗法(IST)消除抑制因子,二是通过止血治疗控制出血。Emicizumab是一种双特异性抗体,可作为因子VIIIa(FVIIIa)的模拟物,在被批准用于先天性A型血友病(cHA)治疗后,在AHA中的应用日益广泛。本综述概述了 AHA 的流行病学、病理生理学、诊断和治疗。对登记、试验和病例系列数据进行了吸收和总结,重点强调了整合使用埃米珠单抗的标准化方法。最近的登记数据表明,有必要关注 AHA 中与免疫抑制相关的死亡率,因此我们以算法的形式提供了治疗建议,这些建议已成为我们医院的标准治疗方法。这些建议旨在最大限度地减少止血产品的使用和与 IST 相关的潜在毒性,同时降低出血的发病率和再住院率。建议的治疗算法包括按治疗阶段划分的关键干预措施,可在使用单阶段检测法快速获取血浆 FVIII 活性的中心随时实施。本文介绍了一个病例,以说明建议的诊断和管理注意事项。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Journal of Thrombosis and Haemostasis
Journal of Thrombosis and Haemostasis 医学-外周血管病
CiteScore
24.30
自引率
3.80%
发文量
321
审稿时长
1 months
期刊介绍: The Journal of Thrombosis and Haemostasis (JTH) serves as the official journal of the International Society on Thrombosis and Haemostasis. It is dedicated to advancing science related to thrombosis, bleeding disorders, and vascular biology through the dissemination and exchange of information and ideas within the global research community. Types of Publications: The journal publishes a variety of content, including: Original research reports State-of-the-art reviews Brief reports Case reports Invited commentaries on publications in the Journal Forum articles Correspondence Announcements Scope of Contributions: Editors invite contributions from both fundamental and clinical domains. These include: Basic manuscripts on blood coagulation and fibrinolysis Studies on proteins and reactions related to thrombosis and haemostasis Research on blood platelets and their interactions with other biological systems, such as the vessel wall, blood cells, and invading organisms Clinical manuscripts covering various topics including venous thrombosis, arterial disease, hemophilia, bleeding disorders, and platelet diseases Clinical manuscripts may encompass etiology, diagnostics, prognosis, prevention, and treatment strategies.
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