a Hemostasis and Thrombosis Center, Clinical Coagulation Laboratory , Children's Hospital Los Angeles , Los Angeles , CA , USA.
b Department of Pediatrics , Keck School of Medicine of the University of Southern California , Los Angeles , CA , USA.
Expert Rev Hematol. 2018 Nov;11(11):835-846. doi: 10.1080/17474086.2018.1531701. Epub 2018 Oct 10.
Hemophilia is a serious bleeding disorder characterized by repeated bleeding episodes into joints and muscles which can lead to permanent disabilities. Treatment with factor replacement therapy has proven to be effective at preventing these complications; however, it can lead to formation of neutralizing antibodies termed inhibitors which significantly complicate the management of the disorder. These inhibitor patients suffer from increased morbidity and mortality and there has been a major unmet need for novel therapeutic approaches. Recently, one such therapy, emicizumab, has been licensed in the United States. Areas covered: This manuscript contains a detailed discussion of the mechanism of action, the clinical trial development program as well as a review of the benefits and risks of this novel agent. In addition, practical considerations for the use of the agent are also described. Expert commentary: Emicizumab represents a new class of medication for the treatment of hemophilia A which in the past has relied on factor replacement therapy and bypassing agent (alternative factor) therapy. Emicizumab fulfills two major unmet needs in patients with hemophilia who have FVIII inhibitors. First, it provides for a much more effective therapy for the prevention of bleeding and second it substantially reduces the treatment burden.
血友病是一种严重的出血性疾病，其特征是反复出现关节和肌肉出血，可导致永久性残疾。用因子替代疗法治疗已被证明能有效预防这些并发症；然而，它会导致中和抗体（称为抑制剂）的形成，这显著增加了疾病管理的复杂性。这些抑制剂患者的发病率和死亡率增加，对新的治疗方法存在重大未满足的需求。最近，一种这样的治疗药物——emicizumab，已在美国获得许可。
本文详细讨论了emicizumab 的作用机制、临床试验开发计划，以及对这种新型药物的益处和风险的回顾。此外，还描述了使用该药物的实际注意事项。
emicizumab 代表了一种治疗 A 型血友病的新型药物，过去依赖于因子替代疗法和旁路（替代因子）疗法。emicizumab 满足了患有 FVIII 抑制剂的血友病患者的两个主要未满足的需求。首先，它为预防出血提供了更有效的治疗方法，其次它大大减轻了治疗负担。
