# 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

## TL;DR

This study evaluates the impact of von Willebrand disease on the quality of life of French adults, revealing significant physical, emotional, and social challenges, especially in severe cases and among women.

## Contribution

The study provides the first prospective evaluation of health-related quality of life in French von Willebrand disease patients using both generic and disease-specific tools.

## Key findings

- Patients with von Willebrand disease show significantly reduced mental and social functioning compared to the general population.
- Severe disease types and women are particularly affected in terms of physical activity, emotional well-being, and future outlook.
- Social health declines over time, and daily life activities like work and leisure are notably impacted.

## Abstract

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.

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).

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.

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.

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.

•HRQoL evaluation in French VWD adults is lacking.•The prospective Willebrand study on HRQoL (WiSH-QoL) is using generic and disease-specific patient-reported outcomes.•VWD reduces physical and mental HRQoL, with an increasing burden for severe types and in women.•VWD impacts adults’ daily life, including work, sport, and leisure and view of future life.

HRQoL evaluation in French VWD adults is lacking.

The prospective Willebrand study on HRQoL (WiSH-QoL) is using generic and disease-specific patient-reported outcomes.

VWD reduces physical and mental HRQoL, with an increasing burden for severe types and in women.

VWD impacts adults’ daily life, including work, sport, and leisure and view of future life.