Functional Assessment in Haemophilia Care: Insights from an Expert Panel on the Use and Applicability of Functional Tests in Clinical Practice

Introduction Recent advances in haemophilia A therapy have markedly improved outcomes and reduced bleeding rates. Consequently, the annual bleeding rate (ABR) alone no longer adequately reflects overall therapeutic success or patient function. Individuals may remain free of bleeding yet experience joint dysfunction, pain, or reduced physical performance. Functional tests, both objective and patient-reported, increasingly complement traditional endpoints by capturing therapy effects on daily life, physical activity, and general health. To support a more comprehensive evaluation of treatment outcomes, an interactive dashboard summarising key functional assessment tools was developed. Method An expert panel of 7 haemophilia specialists met to define general requirements for a functional scoring system and to review the tests included in the dashboard. Experts first outlined the essential characteristics such a system should fulfil (e.g., validity, feasibility, patient relevance). Subsequently, the tests compiled in the dashboard were assessed regarding awareness and utilisation in the experts’ institutions. In a final step, each test was classified according to its potential clinical applicability (diagnostic use, follow-up, rehabilitation, or expert evaluation). A follow-up survey will quantify familiarity and use of the tests across haemophilia centres. Results Experts confirmed that integrating functional assessment tools is essential to complement conventional clinical endpoints in haemophilia management. A key requirement identified was that functional tests should be haemophilia-specific and sensitive to disease-related functional limitations. While some instruments (e.g., the Haemophilia Joint Health Score or the Hemophilia Activities List) were widely used, others—particularly performance-based tests—were less established in routine care. The evaluation revealed differences in applicability between adult and paediatric populations, with limited feasibility reported for younger patients. The systematic classification suggested four major domains for grouping the tests: assessment of joint health, evaluation of quality of life, and a shared intersection covering pain as well as mobility and physical activity. The dashboard and detailed results of this classification will be presented at the congress. Conclusion The expert discussion highlighted the growing importance of functional testing in modern haemophilia care. The upcoming survey will provide further insights into awareness and practical use of functional tools across centres, supporting efforts to enhance patient-centred outcome assessment. Publication History Article published online: 17 February 2026 © 2026. Thieme. All rights reserved. Georg Thieme Verlag KG Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany