ABSTRACT

Introduction

Emicizumab is a bispecific antibody exerting cofactor function of FVIIIa irrespective of the presence of FVIII inhibitors. Long-term data of phase 1/2 and phase 3 studies have been accumulated. Various questions such as indicated patients, ITI, application to PUPs, hemostatic treatment including surgeries, and emicizumab-related morbidity remain to be solved.

Areas covered

The review describes the mode of action, data from pre-/post-marketing and ongoing clinical studies according to PubMed search and our own works.

Expert opinion

For patients with a persistent inhibitor, emicizumab is a definite therapeutic option, although the possibility of BPAs-associated thromboembolic/TMA events raises concerns. The use of ITI together with emicizumab prophylaxis is being examined in clinical trials. For non-inhibitor, especially pediatric patients, emicizumab prophylaxis can be an option. Outcome assessment ‘beyond ABR’ such as joint health, physical/mental activity, QOL is required. Furthermore, continuous data collection for emicizumab-related adverse events and morbidity would be recommended.

中文翻译：

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用于治疗血友病 A 的双特异性抗体

摘要

介绍

Emicizumab 是一种双特异性抗体，无论是否存在 FVIII 抑制剂，都发挥 FVIIIa 的辅因子功能。已经积累了1/2期和3期研究的长期数据。各种问题，如适应症患者、ITI、PUP 的应用、止血治疗（包括手术）和 emicizumab 相关的发病率仍有待解决。

涵盖的领域

根据 PubMed 搜索和我们自己的工作，该审查描述了行动模式、来自上市前/上市后和正在进行的临床研究的数据。

专家意见

对于具有持续抑制剂的患者，emicizumab 是一种明确的治疗选择，尽管 BPA 相关的血栓栓塞/TMA 事件的可能性引起了人们的关注。临床试验中正在检查 ITI 和 emicizumab 预防的使用。对于非抑制剂，尤其是儿科患者，emicizumab 预防可能是一种选择。需要“超越 ABR”的结果评估，例如关节健康、身体/心理活动、生活质量。此外，建议持续收集 emicizumab 相关不良事件和发病率的数据。