Unmet Needs of Patients With Severe Von Willebrand Disease and Their Caregivers: A Qualitative Study on Current Standard of Care in Canada

OBJECTIVES: Von Willebrand disease (VWD) is a hereditary bleeding disorder linked to a deficiency of von Willebrand factor. The impact of living with VWD and the associated burden of disease management is poorly understood. This study aimed to examine key areas of unmet needs and explore the experiences of patients with severe VWD and their caregivers in Canada.

METHODS: A qualitative study using constant comparison technique for thematic analysis targeted adult patients with severe VWD (self-BAT > 10) and their caregivers. Eligible participants underwent a semi-structured telephone interview and were invited to participate in a virtual focus group. Themes covered included the impact of VWD on daily life, satisfaction with existing therapies, disease management, and new treatment expectations.

RESULTS: The study included 10 VWD patients (7 females, 3 males) and 2 caregivers. Among patients, 7 reported that VWD negatively impacted their quality of life, particularly due to physical health limitations. Although the majority (7 out of 10) were satisfied with current prophylaxis treatments, 3 patients feared the potential lack of bleed control and the risk of plasma contamination. Half of the patients experienced adverse events and difficulties with product storage and handling. Seven patients (6 females, 1 male) and both caregivers expressed mental health burdens, including stress and anxiety. Females were disproportionately impacted by VWD-related menorrhagia. A common theme among participants was the need for improved education and awareness of VWD among healthcare professionals and the public. Seven patients expressed the need for improved and less invasive treatment administration, while caregivers cited the need for increased support services in accessing healthcare.

CONCLUSIONS: VWD poses significant burdens on the physical health, quality of life, and well-being of patients and caregivers. There is an opportunity to improve available treatment options and optimize care.