PB0280 - Patient perspective of the burden and severity of von Willebrand disease

Sunday, June 23, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Co-Author(s)

Calvin B. van Kwawegen, MSc

PhD candidate
Erasmus MC, University Medical Centre Rotterdam, Department of Haematology, Rotterdam, The Netherlands
Rotterdam, Zuid-Holland, Netherlands

Presenting Author(s)

Frank W.G Leebeek, MD PhD

Internist-hematologist, Professor
Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands
Rotterdam, Zuid-Holland, Netherlands

Co-Author(s)

SS

Saskia E.M. Schols

Internist-hematologist
Department of Hematology, Radboud university medical center, Nijmegen, The Netherlands
Nijmegen, Gelderland, Netherlands
MC

Marjon H. Cnossen

MD, PhD, professor
Department of Pediatric Hematology and Oncology, Erasmus MC Sophia Children’s Hospital, University Medical Center Rotterdam, Rotterdam, The Netherlands
Rotterdam, Zuid-Holland, Netherlands
Karina Meijer

MD, PhD, professor
Department of Hematology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands
Groningen, Groningen, Netherlands
Ferdows Atiq, MD, MSc, PhD

Senior Research Fellow
Royal College of Surgeons in Ireland
Dublin, Dublin, Ireland
Jeroen Eikenboom

MD, PhD, prof
Department of Thrombosis and Hemostasis, Leiden University Medical Center, Leiden, The Netherlands
Leiden, Zuid-Holland, Netherlands
Kv

Karin van Galen

Internist-Hematologist
Department van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht.
Utrecht, Utrecht, Netherlands
KF

Karin Fijnvandraat

Professor
Emma Kinderziekenhuis/AMC, University of Amsterdam, Netherlands
Amsterdam, Noord-Holland, Netherlands
JD

Joke De Meris

Member workgroup Health and Research
Netherlands Hemophilia Society
Den Haag, Zuid-Holland, Netherlands
AV

Ankse Van der Bom

Professor
Department of Clinical Epidemiology, Leiden University Medical Center, Leiden.
Leiden, Zuid-Holland, Netherlands

Background: Despite many insights on the clinical phenotype of von Willebrand disease (VWD), VWD guidelines prompt additional studies to quantify disease burden as experienced by VWD patients.

Aims: We aimed to investigate patient perspectives on VWD severity, identify determinants of experienced disease burden, and compare patient-reported disease severity with various VWD severity classifications based on factor VIII and von Willebrand factor activity (VWF:Act) levels.

Methods: Patients recruited in the nationwide cross-sectional VWD study in the Netherlands (WiN-study) filled in an extensive questionnaire on the experienced severity of VWD (4-point scale; Table 1), bleeding score (BS) and quality of life (QoL,SF-36). Determinants of patient-reported severity were identified by logistic regression after binarizing the patient-reported classification. We assessed agreement between patient-reported severity and literature-derived severity classifications using Cohen's kappa, a metric commonly employed to quantify concordance in intra- or interobserver evaluations.

Results: We included 736 VWD patients with median age 41.0 (IQR 23.0 – 55.0) years (Table 1). Type 3 patients did not report negligible severity and, compared to the other types, a higher proportion of patients (42.9%,p < 0.01) considered their disease severe (Figure 1A). Higher patient-reported disease severity was associated with lower factor levels, higher bleeding scores and lower physical QoL (Figure 1B-D). Patient-reported severity was independently associated with type 3 (OR=4.10,p=0.025), higher BS (OR=1.09,p < 0.01), and hemostatic treatment in the previous year (OR=1.54,p=0.042). Historical VWF:Act levels with a 0.30IU/mL cutoff as a proxy for severity of VWD aligned strongest with patient-reported severity classification with an accuracy of 64%.

Conclusion(s): We demonstrated that the bleeding phenotype is the most important determinant influencing the perceived severity of VWD from the patient's perspective. Disease severity classification based on VWF levels did not align with the disease severity perceived by patients when taking the bleeding phenotype into account. This underscores the importance of preventing and treating bleeding episodes adequately.