Health-related quality of life in adults with von Willebrand disease: results of the French real-life Willebrand study on health-related quality of life

Authors: Borel-Derlon, A; Veyradier, A; Repessé, Y; Itzhar-Baïkan, N; Desprez, D; Genre-Volot, F; Chambost, H; Ardillon, L; Pan-Petesch, B; Bayart, S; Barthez-Toullec, M; Marin, G; André-Bonnet, MH; Susen, S; Mackensen, S von; Goudemand, J

Affiliations: Hematology and Hemostasis Department, French Reference centre for von Willebrand disease, Haemophilia Treatment Centre, Caen, France. Hematology Department, French Reference centre for von Willebrand disease, Haematology Unit, Lariboisière Hospital, Paris, France. Hematology Department, Haemophilia Treatment Centre, Strasbourg, France. Hematology Department, Haemophilia Treatment Centre, Dijon, France. Pediatric Hematology and Oncology Department, Haemophilia Treatment Centre, Marseille, France. Hematology Department, Haemophilia Treatment Centre, Tours, France. Hematology Department, Haemophilia Treatment Centre, Brest, France. Pediatric Hematology Department, Haemophilia Treatment Centre, Rennes, France. Department of Clinical Development and Medical Affairs, Laboratoire français du Fractionnement et des Biotechnologies (LFB), Les Ulis, France. Department of Statistics, Aixial, Boulogne-Billancourt, France. Hematology and Transfusion Department, French Reference centre for von Willebrand disease, Haemophilia Treatment Centre, Lille, France. Department of Medical Psychology, University Medical Centre, Hamburg-Eppendorf, Germany.

Publication: Research and practice in thrombosis and haemostasis; 2026; 10. 103324

ABSTRACT: BACKGROUND Hemorrhagic events in von Willebrand disease (VWD) impair patients’ physical health, daily functioning, and psychological/emotional well-being. While few studies have assessed health-related quality of life (HRQoL) in VWD, no prospective evaluation had been conducted in France. OBJECTIVES The Willebrand study on HRQoL (WiSH-QoL) is an observational and prospective study that addressed this gap. Conducted in 27 French VWD treatment centers, it employed both generic and VWD-specific patient-reported outcome measures (PROs). METHODS Eligible patients included all ages and VWD types (type 1 restricted to basal von Willebrand factor antigen < 30 IU/dL). PROs (SF-36, VWD-QoL, and VWD-SAT) were assessed at baseline and 24 months. RESULTS In total, 224 adult patients were enrolled. Compared with the French general population, participants showed significantly reduced mental/emotional health and social/physical functioning. The VWD-specific PROs confirmed substantial physical impact in severe disease, including limitations in sports, leisure, and work. They also identified social impacts related to self-perception and relationships (family, others, and professionals). Physical and emotional well-being was particularly affected in women. Regardless of VWD type, patients reported mental health impacts, notably concerning future outlook. Social health deteriorated over time. CONCLUSION The Willebrand study on HRQoL, using disease-specific PROs, reveals the real-life physical, emotional, and social burden of VWD, notably in severe forms and among women. By selecting key questions from these tools, clinicians can better assess these impacts across all patients and provide more comprehensive, long-term support for their well-being.