Department of Molecular Medicine and Haematology, National Health Laboratory Service, Charlotte Maxeke Johannesburg Academic Hospital and School of Pathology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.
S Afr Med J. 2022 May 31;112(6):405-408.
Randomised controlled clinical trial evidence on prophylaxis as optimal care for patients with haemophilia was generated more than a decade ago. However, this knowledge has not translated into clinical practice in South Africa (SA) owing to many barriers to prophylaxis. These include the high treatment burden imposed by prophylaxis (frequent injections two to four times a week), the need for intravenous access to administer replacement clotting factor therapies, and the higher volume of clotting factor required compared with episodic treatment. The recently introduced non-factor therapies in haemophilia care have addressed many of these barriers. For example, emicizumab, which is currently the only globally approved non-factor therapy, can be administered subcutaneously less frequently (weekly, fortnightly or every 4 weeks) and has led to global adoption of prophylaxis as the standard of care in haemophilia by the bleeding disorders community. Haemophilia A is the most prevalent clotting factor deficiency in SA, with >2 000 people diagnosed to date. However, only a few of these patients are currently on prophylaxis. In this 'In Practice' article, we review the rationale for prophylaxis, outline its goals and benefits, and provide evidence-based guidance on which haemophilia patients should be prioritised for emicizumab prophylaxis. This consensus guidance facilitates the adoption of prophylaxis as a national policy and the new standard of care in haemophilia in SA.
十多年前，就已经有了关于预防治疗作为血友病患者最佳护理的随机对照临床试验证据。然而，由于预防治疗存在许多障碍，这种知识并未在南非（SA）的临床实践中得到体现。这些障碍包括预防治疗带来的高治疗负担（每周需要进行两到四次的频繁注射）、需要静脉通路来给予替代凝血因子治疗，以及与偶发性治疗相比需要更多的凝血因子。最近在血友病护理中引入的非因子治疗方法已经解决了其中的许多障碍。例如，emicizumab 是目前唯一获得全球批准的非因子治疗药物，其给药频率较低（每周、每两周或每四周一次），这导致了全球出血性疾病社区将预防治疗作为血友病护理的标准。在 SA，血友病 A 是最常见的凝血因子缺乏症，迄今为止已确诊超过 2000 人。然而，目前只有少数患者接受预防治疗。在这篇“实践”文章中，我们回顾了预防治疗的原理，概述了其目标和益处，并提供了基于证据的指导，说明哪些血友病患者应优先接受 emicizumab 预防治疗。这一共识指南有助于在 SA 将预防治疗作为国家政策和血友病新的护理标准。
