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.

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.