Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2025 Jul;31(4):607-616.
doi: 10.1111/hae.70015. Epub 2025 Apr 17.

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

Yesim Dargaud  1 , Fabienne Volot  2 , Stephanie Desage  1 , Claire Pouplard  3 , Valerie Chamouard  1 , Anne Lienhart  1
Affiliations
Review

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

Yesim Dargaud et al. Haemophilia. 2025 Jul.

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.

Keywords: efanesoctocog alfa; gene therapy; haemophilia; non‐factor therapies; personalized prophylaxis.

PubMed Disclaimer

Conflict of interest statement

The authors declare no conflicts of interest.

Figures

FIGURE 1
FIGURE 1
Clinical, laboratory and environmental variables to take into account for personalized treatment choice.
FIGURE 2
FIGURE 2
Criteria influencing the personalized choice of the prophylactic agent in previously treated haemophilia patients.
FIGURE 3
FIGURE 3
Optimizing prophylactic treatment for haemophilia A patients with and without inhibitors through individualized adjustments.
FIGURE 4
FIGURE 4
Optimizing prophylactic treatment for haemophilia B patients with and without inhibitors through individualized adjustments.
See this image and copyright information in PMC

References

    1. Berntorp E. and Shapiro A. D., “Modern Haemophilia Care,” Lancet 379, no. 9824 (2012): 1447–1456. - PubMed
    1. Srivastava A., Santagostino E., Dougall A., et al., “WFH Guidelines for the Management of Hemophilia,” Haemophilia 26, no. S6 (2020): 1–158. - PubMed
    1. Oldenburg J., Mahlangu J. N., Bujan W., et al., “The Effect of Emicizumab Prophylaxis on Health‐Related Outcomes in Persons With Haemophilia A With Inhibitors: HAVEN 1 Study,” Haemophilia 25, no. 1 (2019): 33–44. - PubMed
    1. Mancuso M. E., Croteau S. E., and Klamroth R., “Benefits and Risks of Non‐Factor Therapies: Redefining Haemophilia Treatment Goals in the Era of New Technologies,” Haemophilia 30, no. S3 (2024): 39–44. - PubMed
    1. Ay C., Frenzel L., Pinachyan K., and Le Quellec S., “Gene Therapy for Haemophilia A and B, From Basic Principles to Clinical Implementation: An Illustrated Review,” Haemophilia 30, no. 1 (2024): 5–15. - PubMed