Mahlangu Johnny N
Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital, School of Pathology, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, 7 York Road, Parktown, Johannesburg 2193, South Africa.
Ther Adv Hematol. 2018 Oct 5;9(11):335-346. doi: 10.1177/2040620718802606. eCollection 2018 Nov.
Whilst the global prevalence of haemophilia B is less than that of haemophilia A, rapid and remarkable innovations have been made in the development of haemophilia B therapies in the last decade. The most recent developments are the evolution of extended half-life haemophilia B replacement therapies which are designed to reduce the treatment burden associated with prophylactic infusion of factor IX (FIX) to prevent bleeding in haemophilia B participants. Clinical development programmes have culminated in the completion of three phase III studies on extended half-life (EHL) recombinant FIX (rFIX) products and subsequent approval and registration of these in many countries around the world. Current data from the three EHL rFIX clinical studies indicate that these products have acceptable safety profiles with no allergic reactions, thromboembolic phenomena or neutralizing antibodies when given to previously treated adolescent and adults for the prevention of bleeds, for the treatment of bleeds and in the perisurgical haemostasis use. Studies in previously untreated paediatric participants are currently ongoing. The EHL rFIX products have the potential impact to reduce the treatment burden associated with prophylactic infusion of replacement FIX, to treat and prevent bleeds in participants with haemophilia B and to improve the participant's health-related quality of life. The impact of EHL rFIX is likely to be modified by current development of other haemophilia B therapy such as antitissue factor pathway inhibitors and haemophilia B gene therapy. In this review, we aim to provide an update on the safety and efficacy data from the three EHL rFIX clinical studies and to consider their roles in the face of novel haemophilia B therapy currently evolving.
虽然全球乙型血友病的患病率低于甲型血友病，但在过去十年中，乙型血友病治疗方法的开发取得了迅速而显著的创新。最新进展是长效乙型血友病替代疗法的发展，其旨在减轻与预防性输注凝血因子IX（FIX）相关的治疗负担，以防止乙型血友病患者出血。临床开发项目最终完成了三项关于长效重组FIX（rFIX）产品的III期研究，并随后在世界许多国家获得批准和注册。来自三项长效rFIX临床研究的当前数据表明，这些产品具有可接受的安全性，在给予既往接受过治疗的青少年和成人用于预防出血、治疗出血以及围手术期止血时，没有过敏反应、血栓栓塞现象或中和抗体。目前正在对既往未接受治疗的儿科患者进行研究。长效rFIX产品有可能减轻与预防性输注替代FIX相关的治疗负担，治疗和预防乙型血友病患者的出血，并改善患者与健康相关的生活质量。长效rFIX的影响可能会因目前其他乙型血友病治疗方法（如抗组织因子途径抑制剂和乙型血友病基因治疗）的发展而改变。在本综述中，我们旨在提供三项长效rFIX临床研究的安全性和有效性数据的最新情况，并考虑它们在当前正在发展的新型乙型血友病治疗方法面前所起的作用。
