Children's Hospital Los Angeles, Hemostasis and Thrombosis Center, Cancer and Blood Disease Institute, Los Angeles, CA, USA.
Department of Hematology and Oncology, University of Southern California, Keck School of Medicine, Los Angeles, CA, USA.
Expert Opin Emerg Drugs. 2021 Dec;26(4):337-350. doi: 10.1080/14728214.2021.1988073. Epub 2021 Oct 11.
Hemophilia is a lifelong, genetic-bleeding disorder, which inadequately treated results in permanent joint damage. It is characterized by spontaneous and trauma-related bleeding episodes. In the last 50 years, treatment has seen dramatic improvements which have improved the quality of life of persons with hemophilia.
This review will provide a summary of current pharmacological approaches for hemophilia A as well as discuss novel agents which are either approved recently or in phase II-III clinical trials, plasma-derived and recombinant factor VIII (FVIII) products, extended half-life FVIII products, bypassing agents and non-replacement therapies.
Novel therapies are already changing the way that hemophilia A is managed, and as more new therapies get approved, there will be a revolution in the management of this serious condition. Clinicians will have both the opportunities as well as the challenges of incorporating such new technologies into clinical practice.
血友病是一种终身遗传性出血性疾病，如果治疗不当，会导致永久性关节损伤。其特征是自发性和创伤相关的出血事件。在过去的 50 年里，治疗方法有了显著的改善，提高了血友病患者的生活质量。
本篇综述将概述目前用于治疗甲型血友病的药物治疗方法，并讨论最近或处于 II-III 期临床试验阶段的新型药物，包括血浆源性和重组凝血因子 VIII（FVIII）产品、半衰期延长的 FVIII 产品、旁路药物和非替代疗法。
新型疗法已经改变了甲型血友病的治疗方式，随着更多新疗法的获批，这种严重疾病的治疗将发生革命性的变化。临床医生将有机会也有挑战将这些新技术融入临床实践。
