Hemostasis and Thrombosis Center and Clinical Coagulation Laboratory, Children's Hospital Los Angeles, Professor of Pediatrics, University of Southern California Keck School of Medicine, Los Angeles, CA, USA.
Br J Haematol. 2019 Aug;186(3):400-408. doi: 10.1111/bjh.15942. Epub 2019 May 9.
Haemophilia is a serious inherited bleeding disorder resulting from a deficiency of coagulation factor VIII (haemophilia A) or coagulation factor IX (haemophilia B). While effective therapies have been developed to replace the missing factor and restore normal coagulation, they can lead to the development of neutralizing anti-drug antibodies, termed inhibitors, which significantly worsen morbidity and quality of life. While advancements have been made to improve the management of patients, and particularly for this review, children with inhibitors, there remain significant unmet needs including inhibitor prevention, inhibitor eradication and, when those fail, more effective bleed prevention and a reduction in the treatment burden. This review will discuss current treatment options including the recently licensed bispecific antibody, emicizumab, and provide insights into how I treat children with inhibitors.
血友病是一种严重的遗传性出血性疾病,由凝血因子 VIII(血友病 A)或凝血因子 IX(血友病 B)缺乏引起。尽管已经开发出有效的治疗方法来替代缺失的因子并恢复正常的凝血功能,但这些治疗方法可能会导致中和性抗药物抗体(称为抑制剂)的产生,从而显著加重发病率和生活质量。尽管在改善患者管理方面取得了进展,特别是对于本综述中的抑制剂患儿,仍然存在重大未满足的需求,包括抑制剂预防、抑制剂清除,以及当这些方法失败时,更有效地预防出血和减轻治疗负担。本文将讨论当前的治疗选择,包括最近获得批准的双特异性抗体emicizumab,并提供我如何治疗抑制剂患儿的见解。