Dargaud Yesim, Volot Fabienne, Desage Stephanie, Pouplard Claire, Chamouard Valerie, Lienhart Anne
National Reference Centre of Haemophilia, Clinical Haemostasis Unit, Hospices Civils de Lyon and Université Claude Bernard Lyon 1, Lyon, France.
Haemophilia Comprehensive Care Centre, Dijon Bourgogne University Hospital, Dijon, France.
Haemophilia. 2025 Jul;31(4):607-616. doi: 10.1111/hae.70015. Epub 2025 Apr 17.
Recent advancements in haemophilia treatment have introduced a range of innovative therapies, including activated FVIII mimetics, ultra-extended half-life recombinant FVIII, rebalancing agents and gene therapy. These developments have transformed treatment options and expanded prophylaxis strategies.
This article reviews key criteria and personalization strategies for the optimization of prophylaxis, tailored to the unique needs of each haemophilia patient on an individual basis.
Although these new therapies offer significant promise in controlling bleeding and enhancing quality of life, challenges such as variable efficacy, potential long-term risks and high costs remain. Additionally, the absence of validated surrogate markers for non-factor therapies limits their optimal use in clinical settings. The primary goal of haemophilia care is to provide optimal personalized treatment that enables patients to lead unrestricted lives. However, the introduction of these novel treatments necessitates a shift in treatment paradigms and the development of improved evaluation tools for better personalization. Choosing the most appropriate treatment requires careful clinical follow-up, taking into account patient preferences, disease burden, and individual and environmental factors that affect patients' lives. Ongoing research and real-world studies are essential to assess long-term efficacy and safety. In addition, improving educational resources-such as digital platforms, virtual reality tools and personalized educational materials tailored to patients' life goals-and effective patient engagement strategies will be critical to achieving successful treatment personalization and adherence.
血友病治疗的最新进展引入了一系列创新疗法，包括活化FVIII模拟物、超长效重组FVIII、再平衡剂和基因疗法。这些进展改变了治疗选择并扩展了预防策略。
本文回顾了优化预防的关键标准和个性化策略，这些策略是根据每位血友病患者的独特需求量身定制的。
尽管这些新疗法在控制出血和提高生活质量方面具有巨大潜力，但仍存在疗效不一、潜在长期风险和高成本等挑战。此外，非因子疗法缺乏经过验证的替代标志物限制了它们在临床环境中的最佳应用。血友病护理的主要目标是提供最佳的个性化治疗，使患者能够过上不受限制的生活。然而，这些新疗法的引入需要转变治疗模式，并开发改进的评估工具以实现更好的个性化。选择最合适的治疗方法需要仔细的临床随访，同时考虑患者的偏好、疾病负担以及影响患者生活的个人和环境因素。持续的研究和真实世界研究对于评估长期疗效和安全性至关重要。此外，改善教育资源，如数字平台、虚拟现实工具以及根据患者生活目标量身定制的个性化教育材料，以及有效的患者参与策略，对于实现成功的治疗个性化和依从性至关重要。
