PB0827 - A Systematic Review of Acquired von Willebrand Syndrome Associated With Non-Cardiac Diseases: Epidemiology, Disease Burden, and Management

Background: Acquired von Willebrand syndrome (aVWS) is a rare bleeding disorder caused by certain underlying conditions, including neoplasms and autoimmune disorders. The pathogenic mechanism of aVWS varies by primary condition but can result in impaired von Willebrand factor (VWF) function, enhanced VWF clearance, or loss of high molecular weight VWF multimers.

Aims: To describe the epidemiology, disease burden, and management of non-cardiac-related aVWS.

Methods: A systematic review (PROSPERO 2021 CRD42021241527) of studies/reports published up to 31 January 2021 (no date limit) was performed using MEDLINE, Cochrane Central Register for Controlled Trials, EMBASE, and Web of Science databases utilizing keywords encompassing aVWS, excluding reports on cardiac conditions. Additional searches were performed using trial registries, grey literature (ethos.bl.uk, dart-europe.eu, oatd.org), congresses, and Google Scholar. Identified studies were screened by 2 independent reviewers. Review articles and duplicates were removed, and data from identified publications were extracted.

Results: Of 300 studies included, 58 were cohort studies (total 13,822 patients) and 242 were case series/reports (327 patients); median (range) patient age at aVWS diagnosis was 55 (1–93) and 51 (1.5–90) years, respectively. Blood system-related neoplasms were the most frequent underlying cause (Table 1). Bleeding-related events were reported in 26 cohort studies (Table 2). VWF/factor VIII concentrate use (Table 2) was reported in only 103 patients in cohort studies. 103 deaths were reported in 6 cohort studies (0.7% of total 13,822 patients from 58 cohort studies), with the highest mortality (82/164 patients; 50%) reported in a study of patients with multiple myeloma and related plasma cell disorders.

Conclusion(s): Myeloproliferative and lymphoproliferative neoplasms were the most common non-cardiac conditions associated with aVWS. This systematic review highlights the limited information available on epidemiology, burden of illness, and treatment of non-cardiac-related aVWS, and the need for further research to better understand this rare disease and improve the patients’ unmet needs.