Abstract
INTRODUCTION: Assessment of health-related quality of life (HRQoL) is relatively new in von Willebrand disease (VWD). So far, generic questionnaires have mainly been used for HRQoL assessment in VWD. AIMS: To assess generic and disease-specific HRQoL in adult VWD patients and compare HRQoL with the general German population. METHODS: Patients presenting with a personal or family history of bleeding and von Willebrand factor (VWF)-specific laboratory parameters were enrolled in the WIL-QoL study. HRQoL was assessed with generic (SF-36) and disease-specific (VWD-QoL) questionnaires. Descriptive and inferential statistical procedures were applied based on a significance level at α ≤ 5%. RESULTS: In the retrospective, multicentre WIL-QoL study, HRQoL and clinical data in 120 adults with VWD (one adolescent completed the adult questionnaire) from 10 centres in Germany were collected. Compared to the corresponding age group in the general German population, female VWD patients had significantly worse HRQoL in all SF-36 domains and male patients only in the 'physical functioning' domain. In the VWD-QoL, highest impairments were seen in all VWD patients in the domains 'other physicians', 'treatment' and 'sport & leisure'. VWD patients with a more significant disease burden, such as a bleeding score ≥ 9 (p < 0.0001), long-term prophylaxis (p = 0.003), and VWD-type 3 (p = 0.022), reported significantly worse HRQoL. No HRQoL differences were seen between male and female VWD patients. CONCLUSION: Female VWD patients showed significant impairments in their HRQoL compared to the age group-related general population. Compared to SF-36, the VWD-QoL identified stronger significant HRQoL differences in most VWD subgroups, confirming the impact of bleeds.