Presentation Details
| The Severe Von Willebrand Disease Patient Registry: A Longitudinal Natural History and Patient Outcomes Study Johnna Cesta1, Mrinal Gounder1, Christopher Walsh 2, Alice Araphsian1, Jeanette Cesta1, Christina Morgenthaler1. 1VWD Connect Foundation, Wellington, FL, USA.2Icahn School of Medicine Mount Sinai Hospital, New York, NY, USA |
Abstract
Background: Severe von Willebrand Disease (sVWD) is a rare bleeding disorder with a natural history poorly defined in literature. VWD Connect Foundation (VCF) is a U.S. National non-profit patient advocacy organization serving the sVWD patient community. To address the research gaps, VCF initiated a prospective study titled “The Severe Von Willebrand Disease Patient Registry: A Longitudinal Natural History and Patient Outcomes Study” (IRB VWD-001). Objectives: The primary objective is to characterize the sVWD patient population through the use of an online portal where patient demographics, medical history, self-reported bleeding history and impact on quality of life are investigated. This data will provide researchers and other stakeholders with natural history data on the sVWD population to set as benchmarks to develop guidelines and guide treatment and clinical trial design. Methods: Enrollment criteria include a patient diagnosis of Type 3 VWD or Severe Types 1, 1C, 2A, 2B, 2M, and 2N, excluding acquired vWD. Patients with unknown/other types may be considered severe if they have von Willebrand factor levels <20%. Following informed consent and screening, participants complete online modules, with continued participation as new modules are released. Current modules include Demographics and the Self-Administered Bleeding Assessment Tool (Self-BAT; Deforest et al, 2015). Additionally, current medications and medical history data are collected and updated by the participant as needed over time. Future modules planned include: quality of life, family history, laboratory data, genetic data, reproductive bleeding, joints, treatments, prophylaxis and inhibitors. Results: From December 2021 to December 2023, 44 participants have enrolled, and recruitment is ongoing. 40 of the 44 participants are diagnosed Type 3 VWD. Select participant data on demographics, Self-BAT responses, treatment, and medical and medication history will be presented at the meeting. Conclusions: To advance the understanding of severe von Willebrand Disease, robust data describing patients’ medical history and lived experiences must be collected. By gathering broad data from those with sVWD from all over the United States, this Registry is beginning to address this research gap. The Registry’s collection of further information on diagnosis, phenotype, genetic and laboratory data will accelerate this mission.
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No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.