Division of Haematology/Oncology; Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
Hematology Department, Ampang Hospital, Ampang, Selangor, Malaysia.
Haemophilia. 2024 Apr;30 Suppl 3:86-94. doi: 10.1111/hae.14964. Epub 2024 Mar 24.
Prophylaxis has become standard of care for all persons with haemophilia (PWH) with a severe phenotype. However, 'standard prophylaxis' with either factor or non-factor therapies (currently only emicizumab available) is prohibitively expensive for much of the world. We sought to address the question of 'How much prophylaxis is enough?' and 'Can it be individualized?' and specifically 'Can emicizumab be individualized?'.
We reviewed the literature on prophylaxis in haemophilia since its inception in the 1950s to the present, the development of more and less intense factor prophylaxis regimens and their outcomes and additionally the published outcomes of prophylaxis with low dose emicizumab.
What these experiences collectively show is that low dose emicizumab does result in significant benefits to patients whilst being much less expensive than a "one size fits all" emicizumab prophylaxis approach. We also took note that some non-factor therapies still in development are individualized given that high doses of these can potentially put patients at risk.
Prophylaxis is now clearly accepted as standard of care for PWH with a severe phenotype but now in a very short time a large assortment of different treatment options for prophylaxis have become/are becoming available and the haemophilia community will need to determine how to best use these recognizing that no 'one treatment fits all'.
预防治疗已成为所有严重血友病患者（PWH）的标准治疗方法。然而，对于世界上大多数地区来说，使用因子或非因子治疗（目前仅可使用emicizumab）进行“标准预防治疗”费用过高。我们试图回答以下问题：“需要进行多少预防治疗？”和“是否可以个体化？”，具体而言，“可以个体化使用 emicizumab 吗？”。
我们回顾了自 20 世纪 50 年代预防治疗开始以来的文献，包括更强化和非强化因子预防治疗方案的发展及其结果，以及低剂量 emicizumab预防治疗的已发表结果。
这些经验共同表明，低剂量 emicizumab 确实为患者带来了显著益处，同时比“一刀切”的 emicizumab 预防治疗方法成本低得多。我们还注意到，一些仍在开发中的非因子治疗方法也可以个体化，因为这些药物的高剂量可能会使患者面临风险。
预防治疗现在已被明确接受为严重血友病患者的标准治疗方法，但在很短的时间内，大量不同的预防治疗选择已经/正在出现，血友病社区将需要确定如何最好地利用这些治疗方法，认识到没有“一刀切”的治疗方法。
