获得性血友病患者使用 Emicizumab 的疗效:系统回顾

IF 2.3 4区 医学 Q2 HEMATOLOGY
Ghachem Ikbel, Baccouche Hela, Kaabar Mohamed Yassine, Khemiri Hamida, Ben Salem Kamel
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引用次数: 0

摘要

背景:Emicizumab 是一种双特异性 VIII 因子模拟抗体,于 2018 年获批用于有或无抑制剂的先天性 A 型血友病的出血预防。此后,一些病例报告和病例系列描述了在获得性 A 型血友病(AHA)中标签外使用埃米珠单抗的情况,最近还公布了两项临床试验(AGEHA、GTH-AHA-EMI)的数据:目的:描述有关埃米珠单抗疗效的报告数据,强调其在治疗中的获益/风险情况:方法:我们在PubMed、Scopus、Cochrane和Google Scholar上进行了文献检索,包括截至2024年8月所有报道AHA患者使用埃米珠单抗临床疗效的科学文章:32项研究被纳入最终综述,共涉及171名AHA患者。大多数患者开始使用埃米珠单抗是为了积极控制出血,并通过各种方案进行预防。随访时间和缓解标准各不相同。两项临床试验支持使用埃米珠单抗预防出血,并采用了新的给药方案和完成标准。所有病例的出血都得到了很好的控制,没有出现大的复发性出血。报告了一些不良事件:3例深静脉血栓形成,2例中风,2例有血栓栓塞风险因素的患者产生了抗伊米珠单抗药物抗体:根据已发表的数据,埃米珠单抗似乎能有效控制和预防AHA患者的出血,并且具有良好的获益/风险特征。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Outcomes of Emicizumab in Acquired Hemophilia Patients: A Systematic Review.

Background: Emicizumab, a bispecific factor VIII mimetic antibody, was approved in 2018 for bleeding prophylaxis in congenital hemophilia A with or without inhibitors. Since then, several case reports and case series have described the off-label use of emicizumab in acquired hemophilia A (AHA), and data from two clinical trials were recently published (AGEHA, GTH-AHA-EMI).

Objectives: To describe the reported data on the outcomes of emicizumab, highlighting its benefit/risk profile in treatment.

Methods: We conducted a literature search in PubMed, Scopus, Cochrane, and Google Scholar up to August 2024, including all scientific articles reporting clinical outcomes of emicizumab use in patients with AHA.

Results: Thirty-two studies were included in the final review, covering a total of 171 AHA patients. The majority started emicizumab for active bleeding management and prophylaxis with various regimens. Follow-up duration and remission criteria varied. Two clinical trials supported the use of emicizumab for bleeding prophylaxis with a new dosing regimen and completion criteria. Bleeding was well managed in all cases, with no major recurrent bleeds. Some adverse events were reported : 3 cases of deep venous thrombosis, 2 cases of stroke, and 2 cases of anti-emicizumab drug antibodies developing in patients with thromboembolic risk factors.

Conclusions: Based on published data, emicizumab appears to be effective in bleeding management and prophylaxis in AHA patients, with a favorable benefit/risk profile.

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来源期刊
CiteScore
4.40
自引率
3.40%
发文量
150
审稿时长
2 months
期刊介绍: CATH is a peer-reviewed bi-monthly journal that addresses the practical clinical and laboratory issues involved in managing bleeding and clotting disorders, especially those related to thrombosis, hemostasis, and vascular disorders. CATH covers clinical trials, studies on etiology, pathophysiology, diagnosis and treatment of thrombohemorrhagic disorders.
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