Haemophilia Prophylaxis in the Age of Innovation: Exploring Opportunities for Personalized Treatment.

Abstract

Introduction: 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.

Aim: 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.

Discussion and conclusion: 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.