The prognosis of acquired haemophilia A (AHA) is severe and treatment options are limited. Emicizumab is a novel bispecific humanized monoclonal antibody in the treatment of inherited AHA with inhibitors. An 83-year-old AHA patient with congestive heart failure and a high risk for thromboembolic and cardiac events who had initially been treated successfully with steroids and substitution of recombinant B-domain-deleted porcine FVIII developed severe bleeding complications and a secondary increase in inhibitor titres after 4 weeks of treatment. Conventional therapeutic strategies failed, and the patient was subsequently treated with emicizumab on off-label and named patient use premises. After the application of emicizumab, the clinical conditions stabilized and no further substitution of coagulation factors was needed. The patient could be discharged and survived 36 days in a cardiac rehabilitation centre without indications for spontaneous bleeding or thromboembolic events. We suggest that the effects of emicizumab in acquired haemophilia should be evaluated in clinical trials.

中文翻译：

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Emicizumab 治疗获得性血友病：病例报告

获得性血友病 A (AHA) 的预后很严重，治疗选择有限。Emicizumab 是一种新型双特异性人源化单克隆抗体，用于治疗遗传性 AHA 和抑制剂。一名患有充血性心力衰竭、血栓栓塞和心脏事件高风险的 83 岁 AHA 患者，最初用类固醇和重组 B 结构域缺失的猪 FVIII 替代成功治疗，出现严重出血并发症和抑制物继发性增加治疗 4 周后滴度。传统的治疗策略失败了，该患者随后在标签外和指定的患者使用场所接受了 emicizumab 治疗。应用emicizumab后，临床情况稳定，无需进一步替代凝血因子。患者可以出院并在心脏康复中心存活 36 天，没有自发性出血或血栓栓塞事件的指征。我们建议应在临床试验中评估 emicizumab 对获得性血友病的影响。