Characterizing the UK Population Living With Von Willebrand Disease: Foundational Data for Health Economics and Outcomes Research
Author(s)
George Morgan1, Emily Back, BSc, MSc1, Jo Traunter, EdD2, Hannah Yarnall, MSc3, Daryl-Ashlee Preston, BSc4, Sarah Brighton, MSc1, Samuel Bristow, BSc, MSc1.
1DHT.health, Clitheroe, United Kingdom, 2The University of Hull, Hull, United Kingdom, 3The Haemophilia Society, London, United Kingdom, 4NHS Business Service Authority, Newcastle, United Kingdom.
1DHT.health, Clitheroe, United Kingdom, 2The University of Hull, Hull, United Kingdom, 3The Haemophilia Society, London, United Kingdom, 4NHS Business Service Authority, Newcastle, United Kingdom.
OBJECTIVES: von Willebrand Disease (vWD) is a rare, inherited, heterogenous, and under-researched bleeding disorder. An international, direct-to-community study, enables detailed characterisation of this patient community in the United Kingdom (UK), informing health economics and outcomes research (HEOR), particularly in burden of illness, healthcare resource use, and patient preference.
METHODS: Data were drawn from the UK cohort of the PIVOT-vWD study (collected Oct 24-Jan 25), a cross-sectional study co-developed with key opinion leaders within the vWD community to capture the impact, voice, and outcomes of people living with vWD. Invitations were distributed via a network of patient organisations. Participants (patients and/or caregivers) completed an encrypted online questionnaire. Individuals self-reported demographic, clinical, and treatment characteristics, bleed impact, healthcare resource use, outcome measures including quality of life, visual analogue scales (VAS), and treatment-focused preference ranking exercises. Descriptive statistics were used to summarise the cohort.
RESULTS: Sample includes 116 individuals (83% self-complete; 17% proxy-complete), with a mean age of 41.9 years (SD: 21.4), and 66% female. Respondents were geographically dispersed across the UK, with the highest representation from South East England (22%). Self-reported vWD types included: Type 1 (28%), Type 2 including subtypes (51%), Type 3 (10%), Acquired (3%), and 8% Unreported. Treatment use was reported by 69%, and 76% had accessed hospital-based care in the past 12 months. Mean satisfaction with hospital care was 7.2 out of 10 (SD: 2.6) on the VAS.
CONCLUSIONS: This dataset offers a comprehensive, real-world, patient reported resource characterising people living with vWD in the UK, including perspectives from both patients and caregivers. Findings reveal variation in treatment exposure and hospital interaction, with a substantial proportion reporting minimal or no engagement with care. PIVOT-vWD supports future HEOR by enabling robust analysis of service use, patient preference, and burden, grounded in the real-world patient voice of the vWD community.
METHODS: Data were drawn from the UK cohort of the PIVOT-vWD study (collected Oct 24-Jan 25), a cross-sectional study co-developed with key opinion leaders within the vWD community to capture the impact, voice, and outcomes of people living with vWD. Invitations were distributed via a network of patient organisations. Participants (patients and/or caregivers) completed an encrypted online questionnaire. Individuals self-reported demographic, clinical, and treatment characteristics, bleed impact, healthcare resource use, outcome measures including quality of life, visual analogue scales (VAS), and treatment-focused preference ranking exercises. Descriptive statistics were used to summarise the cohort.
RESULTS: Sample includes 116 individuals (83% self-complete; 17% proxy-complete), with a mean age of 41.9 years (SD: 21.4), and 66% female. Respondents were geographically dispersed across the UK, with the highest representation from South East England (22%). Self-reported vWD types included: Type 1 (28%), Type 2 including subtypes (51%), Type 3 (10%), Acquired (3%), and 8% Unreported. Treatment use was reported by 69%, and 76% had accessed hospital-based care in the past 12 months. Mean satisfaction with hospital care was 7.2 out of 10 (SD: 2.6) on the VAS.
CONCLUSIONS: This dataset offers a comprehensive, real-world, patient reported resource characterising people living with vWD in the UK, including perspectives from both patients and caregivers. Findings reveal variation in treatment exposure and hospital interaction, with a substantial proportion reporting minimal or no engagement with care. PIVOT-vWD supports future HEOR by enabling robust analysis of service use, patient preference, and burden, grounded in the real-world patient voice of the vWD community.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR42
Topic
Economic Evaluation, Patient-Centered Research, Study Approaches
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)