Department of Haematology and Transfusion Medicine, "Carlo Poma" Hospital, Mantua, Italy.
Italian National Blood Centre, National Institute of Health, Rome, Italy.
Blood Transfus. 2019 May;17(3):223-228. doi: 10.2450/2019.0026-19.
One of the most serious complications of the treatment of severe haemophilia A is the development of alloantibodies against exogenous factor VIII (FVIII). Inhibitors render factor replacement therapy ineffective, exposing patients to a remarkably high risk of morbidity and mortality. Besides the well-known bypassing agents (i.e. activated prothrombin complex concentrate and recombinant activated factor VII) used to treat or prevent bleeding in haemophilia patients with inhibitors, there is growing interest in newer haemostatic therapies that are not based on the replacement of the deficient FVIII. This review will focus on the most interesting among these innovative therapies, emicizumab, and will provide an update on its current stage of clinical development.
治疗重型 A 型血友病最严重的并发症之一是对外源性因子 VIII (FVIII)产生同种抗体。抑制剂使因子替代疗法无效，使患者面临极高的发病率和死亡率风险。除了用于治疗或预防有抑制剂的血友病患者出血的众所周知的旁路制剂（即激活的凝血酶原复合物浓缩物和重组激活的因子 VII）外，人们对不基于替代缺乏的 FVIII 的新型止血治疗方法越来越感兴趣。本综述将重点介绍这些创新疗法中最有趣的依替巴肽，并提供其当前临床开发阶段的最新信息。
