Von Willebrand Disease Epidemiology, Burden of Illness and Management: A Systematic Review

Abstract

Ping Du,1 Aurore Bergamasco,2 Yola Moride,2,3 Françoise Truong Berthoz,4 Gülden Özen,1 Spiros Tzivelekis1 1Takeda Development Center Americas, Inc., Cambridge, MA, USA; 2YOLARX Consultants, Paris, France; 3Rutgers, The State University of New Jersey, New Brunswick, NJ, USA; 4Takeda Pharmaceuticals International AG, Zürich, SwitzerlandCorrespondence: Ping Du, Takeda Development Center Americas, Inc., 40 Landsdowne Street, Cambridge, MA, 02139, USA, Tel [phone], Email [email protected]: Although hereditary von Willebrand disease (VWD) is the most common bleeding disorder, its epidemiology is not well understood. A systematic review (PROSPERO CRD42020197674/CRD42021244374) on the epidemiology/burden of illness of VWD was conducted to better understand patients’ unmet needs.Methods: Observational studies (published January 1, 2010 to April 14, 2021) were identified in MEDLINE and Embase databases, using free-text keywords and thesaurus terms for VWD and outcomes of interest. Pragmatic web-based searches of the gray literature, including conference abstracts, were performed, and reference lists of retained publications were manually searched for additional sources. Case reports and clinical trials (phase 1– 3) were excluded. Outcomes of interest were incidence, prevalence, mortality, patient characteristics, burden of illness, and therapeutic management/treatments currently used for VWD.Results: Of the 3095 identified sources, 168 were included in this systematic review. Reported VWD prevalence (22 sources) ranged from 108.9 to 2200 per 100,000 in population-based studies and from 0.3 to 16.5 per 100,000 in referral-based studies. Reported times between first symptom onset and diagnosis (two sources; mean 669 days; median 3 years) highlighted gaps in timely VWD diagnosis. Bleeding events reported in 72– 94% of the patients with VWD (all types; 27 sources) were mostly mucocutaneous including epistaxis, menorrhagia, and oral/gum bleeding. Poorer health-related quality of life (three sources) and greater health care resource utilization (three sources) were reported for patients with VWD than in general populations.Conclusion: Available data suggest that patients with VWD experience high disease burden in terms of bleeding, poor quality of life, and health care resource utilization.Keywords: disease burden, epidemiology, health-related quality of life, health care resource utilization, systematic review, von Willebrand disease

Keywords

• disease burden
• epidemiology
• health-related quality of life
• health care resource utilization
• systematic review
• von willebrand disease.