ArticleHaemophilia : the official journal of the World Federation of Hemophilia2024
Patient-reported data on the severity of Von Willebrand disease.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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Who cites it
3 citing papers in PubMed.
- Gynecologic and obstetric management of girls and women with von Willebrand disease.Haematologica · 2026Review
- Article
- Patient-reported data on the severity of Von Willebrand disease.Haemophilia : the official journal of the World Federation of Hemophilia · 2024Article
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Authors and funding
13 authors.
Funding
Abstract
introductionThe severity of Von Willebrand disease (VWD) is currently based on laboratory phenotype. However, little is known about the severity of the patient's experience with the disease. The most recent VWD guidelines highlight the need for patient-reported outcomes (PROs) in VWD.
aimThe study aimed to investigate the patient-perspective on VWD severity and to identify key factors that determine the severity of disease experienced by patients. MATERIALS AND
methodsPatients participated in a nationwide cross-sectional study on VWD in the Netherlands (WiN-study). Patients filled in a questionnaire containing questions on the experienced severity of VWD (4-point scale), bleeding score (BS) and quality of life (QoL).
resultsWe included 736 patients, median age of 41.0 years (IQR 23.0-55.0) and 59.5% were women. A total of 443 had type 1, 269 type 2 and 24 type 3 VWD. Self-reported severity of VWD was categorized as severe (n = 52), moderate (n = 171), mild (n = 393) or negligible (n = 120). Classification by historically lowest FVIII:C levels < 0.20 IU/mL as a proxy for severe VWD aligned with patient-reported severity classification with a 72% accuracy. Type 3 VWD (OR = 4.02, 95%CI: 1.72-9.45), higher BS (OR = 1.09, 95%CI: 1.06-1.11), female sex (OR = 1.36, 95%CI: 1.01-1.83), haemostatic treatment in the year preceding study inclusion (OR = 1.53, 95%CI: 1.10-2.13) and historically lowest VWF:Act levels (OR = 0.26, 95%CI: 0.07-1.00) were independent determinants of patient-reported severity.
conclusionThis study shows that patient-reported data provide novel insights into the determinants of experienced disease severity. Our findings highlight the need for studies on PROs with validated questionnaires to assess the burden of VWD.
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