Department of Hematology and Bone Marrow Transplantation, Poznań University of Medical Sciences, Poznań, Poland.
Expert Rev Hematol. 2022 Oct;15(10):933-942. doi: 10.1080/17474086.2022.2131526. Epub 2022 Oct 16.
Hemophilia A is a genetically conditioned disease leading to hemostatic disorders due to factor VIII (FVIII) deficiency. The treatment of hemophilia has evolved throughout the past years and has significantly changed. One of the newest drugs for prophylactic treatment is the humanized bispecific IgG antibody - emicizumab, which binds with factor IXa and factor X, bridging those factors and thus mimicking the activity of factor VIII.
The literature search was done via the PubMed database, with the emphasis on clinical trials and case reports, describing the off-label emicizumab use. This review presents an extensive summary and considers the advantages and disadvantages (side-effects) of emicizumab, describing additional clinical situations, where emicizumab has been successfully used. In our review, we cover information about the mechanisms of action, indications, and efficacy and discuss some chosen case reports about off-label emicizumab use.
Its convenient administration method (subcutaneous) and frequency of injections (from once a week to once a month) makes it a more comfortable treatment, limiting injection-site reactions, hospital stays, costs of prophylaxis, and significantly increasing patients' quality of life. Adverse effects are scarce and rarely serious - the most common ones are reactions at the injection-site and upper respiratory tract infections.
血友病 A 是一种遗传性疾病，由于因子 VIII（FVIII）缺乏导致止血障碍。血友病的治疗在过去几年中不断发展，并发生了重大变化。最新的预防性治疗药物之一是一种人源化双特异性 IgG 抗体——emicizumab，它可以结合因子 IXa 和因子 X，桥接这些因子，从而模拟因子 VIII 的活性。
通过 PubMed 数据库进行文献检索，重点关注描述emicizumab 标签外使用的临床试验和病例报告。本综述提供了广泛的总结，并考虑了 emicizumab 的优缺点（副作用），描述了 emicizumab 成功应用的其他临床情况。在我们的综述中，我们涵盖了关于作用机制、适应症和疗效的信息，并讨论了一些关于标签外使用 emicizumab 的选定病例报告。
其方便的给药方式（皮下）和注射频率（从每周一次到每月一次）使其成为一种更舒适的治疗方法，减少了注射部位反应、住院时间、预防治疗费用，并显著提高了患者的生活质量。不良反应罕见且通常不严重——最常见的是注射部位反应和上呼吸道感染。
