Abstract Number: VPB0836
Meeting: ISTH 2022 Congress
Background: Severe Von Willebrand Disease (sVWD) patients (estimated over 1500 U.S.) are an under-studied population with severe bleeding not well-characterized in literature. VWD Connect Foundation (VCF) has sponsored the design and launch of a sVWD online longitudinal natural history and outcomes patient registry.
Aims: The primary objective is to characterize the sVWD patient population, reporting on prevalence, genotypes, phenotypes, and management. The secondary objective is to provide a convenient online platform for participants (or caregivers) to self-report clinical outcomes in real time. This critical endeavor also aims to provide researchers and other stakeholders with actionable data describing the sVWD population leading to better treatments, management, and ultimately a cure.
Methods: August 2021: VCF received IRB approval for protocol VWD-001, The Severe Von Willebrand Disease Patient Registry: A Longitudinal Natural History and Patient Outcomes Study. December 2021: the Registry opened to a small cohort of participants. For the purposes of this Registry, sVWD patients may include Type 3 VWD and 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 modules, with continued participation as new modules are released. Future modules planned include: quality of life, family history, laboratory data, genetic data, reproductive bleeding, joints, treatments, prophylaxis and inhibitors. Participants will complete the Self-Administered Bleeding Assessment Tool (Self-BAT; Deforest et al, 2015) annually.
Results: As of January 2022, preliminary data has been collected on a small cohort, with enrollment and participation ongoing. Data includes patient demographics, medical history, prior and concomitant medication history, and Self-BAT results.
Conclusion(s): Using the skill of its membership and dedicated medical professionals, VCF has successfully launched an online sVWD Patient Registry. Preliminary data from a larger cohort would be presented at the meeting.
To cite this abstract in AMA style:Cesta JM, Arapshian A, Cesta J, Gounder M, Walsh C, Morgenthaler C. VWD Connect Foundation Severe Von Willebrand Disease Patient Registry [abstract]. https://abstracts.isth.org/abstract/vwd-connect-foundation-severe-von-willebrand-disease-patient-registry/. Accessed September 27, 2023.
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