Department of Hematology, University El Manar Faculty of medecine of Tunis, Tunis, Tunisia.
Laboratory of Hematology, Taher Maamouri Hospital, Nabeul, Tunisia.
Clin Appl Thromb Hemost. 2024 Jan-Dec;30:10760296241298661. doi: 10.1177/10760296241298661.
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).
To describe the reported data on the outcomes of emicizumab, highlighting its benefit/risk profile in treatment.
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.
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.
Based on published data, emicizumab appears to be effective in bleeding management and prophylaxis in AHA patients, with a favorable benefit/risk profile.
依库珠单抗是一种双特异性因子 VIII 模拟抗体，于 2018 年获批用于有或无抑制剂的先天性 A 型血友病患者的出血预防。此后，多项病例报告和病例系列描述了依库珠单抗在获得性 A 型血友病（AHA）中的超适应证使用，最近发表了两项临床试验的数据（AGEHA、GTH-AHA-EMI）。
描述依库珠单抗的报告数据，重点介绍其治疗中的获益/风险特征。
我们在 PubMed、Scopus、Cochrane 和 Google Scholar 上进行了文献检索，检索截至 2024 年 8 月，包括所有报告依库珠单抗治疗 AHA 患者临床结局的科学文章。
最终综述共纳入 32 项研究，共纳入 171 例 AHA 患者。大多数患者开始使用依库珠单抗进行活动性出血的管理和预防，采用不同的方案。随访时间和缓解标准各不相同。两项临床试验支持依库珠单抗用于出血预防的新剂量方案和完成标准。所有病例的出血均得到良好管理，无重大复发性出血。报告了一些不良事件：3 例深静脉血栓形成，2 例中风，2 例有血栓栓塞危险因素的患者出现抗依库珠单抗药物抗体。
基于已发表的数据，依库珠单抗似乎对 AHA 患者的出血管理和预防有效，具有良好的获益/风险特征。
