Addressing unmet needs in rare bleeding disorders: selected poster extracts of recent research in hemophilia A and von Willebrand disease presented at the 14th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) (Feb 3–5, 2021; virtual congress). (23rd September 2021)

• Record Type: Journal Article
• Authors: Allen, Geoffrey; Du, Ping; Khair, Kate; Lee, Hye-Youn; Ozelo, Margareth C.; Berthoz, Françoise Truong; Windyga, Jerzy
• Is Part Of: Expert review of hematology. Volume 14:Supplement 1(2021)
• Page Start: 1
• Page End: 18
• Publication Date: 2021-09-23
• Subjects: Rare bleeding disorders -- hemophilia A -- factor VIII -- personalized prophylaxis -- von Willebrand disease -- von Willebrand factor -- burden of disease -- unmet need; Hematology -- Periodicals; 616.15005
• DOI: 10.1080/17474086.2021.1963706
• Languages: English
• ISSNs: 1747-4086

Abstract

ABSTRACT: Hemophilia A and von Willebrand disease (VWD) are inherited rare bleeding disorders affecting normal hemostasis. Patients with VWD, especially those with severe disease types, share some similarities to patients with hemophilia A in their burden of disease: they suffer from an increased risk of potentially severe and life-threatening bleeds and associated long-term consequences, such as impaired joint health and overall lower quality of life. However, the two bleeding disorders differ in their primary cause and affected patient population, and comprise a range of different bleeding phenotypes with varying unmet needs. Generating scientific evidence to advance health care for patients with rare bleeding disorders is challenging due to the low prevalence and heterogeneity of affected populations, including patient demographics and symptom severities. Innovative study designs are needed to adequately answer relevant scientific questions and address patients' unmet needs. In support of advancing clinical outcomes and treatment options for these patients, at the recent EAHAD 2021 annual congress, novel approaches and data from clinical and real-world observational studies, as well as systematic literature analyses, were presented. Herein, extracts from seven selected posters reporting research in hemophilia A and VWD funded by Takeda are discussed.