Normalization of Haemostasis in People with Haemophilia A: Expert Consensus on Unmet Needs and a Framework for...

New therapies for haemophilia A have created momentum for enhancing protection against bleeding.

The ultimate objective is to address remaining unmet needs and promote health equity.

In this initiative, 14 haemophilia A experts from Europe ( n  = 13) and North America ( n  = 1) were involved to identify unmet needs in people with haemophilia A (PwHA) and measures to address these needs (e.g., normalization of factor VIII [FVIII] levels) to facilitate health equity. It combined online workshops, a modified Delphi process to develop consensus statements on remaining unmet medical needs (consensus was defined as ≥70% of panellists who gave a rating of 4 [agree] or 5 [strongly agree] using a 5-point Likert scale), and development of policy recommendations.

Among the panellists who voted, consensus was reached on 25/26 statements, including 13 with 100% agreement. The experts outlined a need to optimize prophylaxis in all eligible PwHA, aiming for high-sustained FVIII levels or normalized haemostasis. The panel also highlighted a need to prevent all bleeds (including microbleeds) that can occur despite prophylaxis, and to address chronic pain which is highly prevalent. Overall, 23 policy recommendations were finalized, which included the wider use of prophylaxis to reduce the burden of disease on PwHA and/or provide normalized haemostasis, the expansion of multidisciplinary teams and the establishment of networks of expertise, and sharing of specialist knowledge.

This initiative lays an ambitious foundation to rigorously evaluate unmet needs in PwHA and the determinants of health equity, offering a comprehensive set of recommendations to overcome barriers and achieve the possibility of normalization of haemostasis.