Assessing the Sensitivity of EQ-5D-5L and AQoL-4D to Disease Severity in Von Willebrand Disease: Findings From UK Cohort of the PIVOT-vWD Study
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 bleeding disorder characterised by frequent bleed events impacting health-related quality of life (HRQoL). This research assessed the sensitivity of the EQ-5D and AQoL-4D to assess changes in HRQoL by vWD type.
METHODS: Data were utilised from the UK cohort of the PIVOT-vWD study (Oct 24 - Jan 25); a direct-to-community, cross-sectional study capturing outcomes of vWD across 116 individuals. Respondents reported patient and clinical characteristics, alongside the EQ-5D-5L and AQoL-4D (self and/or proxy-reported versions). Participants were grouped by their self-reported vWD type (Type 1, 2 and 3); those ‘unreported’ or ‘acquired’ were excluded. Distribution of responses across domains of each HRQoL instrument were assessed using frequency and percentage. Spearman’s rank coefficients were calculated to analyse associations between instrument domains.
RESULTS: The included sample totalled 104 individuals (86 self-complete/18 proxy-complete). EQ-5D-5L results were similar for Type 1 and 2, with 27% vs. 25% reporting moderate to extreme problems in mobility, 12% vs. 12% in self-care, and 30% vs. 32% in usual activities. These rose in Type 3 to 83%, 50%, and 67% respectively; pain/discomfort (27-29% vs. 43%) and anxiety/depression (18-20% vs. 43%) also increased. Across 12 AQoL-4D items, responses were similar for Types 1 and 2. Type 3 reported more problems with self-care, mobility, household tasks, family role, and pain. Sleep, anxiety, and friendships showed moderate burden across types; hearing and communication were rarely affected. Convergent correlations were strongest for anxiety (rs=0.87) and pain (rs=0.82). Sensory and communication domains were more distinct to AQoL-4D (rs=<0.33 across EQ-5D domains).
CONCLUSIONS: HRQoL burden was greatest in Type 3 vWD, while responses were broadly similar between Type 1 and 2 across both instruments. While both tools captured physical and emotional impacts, AQoL-4D may offer added value by capturing broader social and sensory domains relevant to quality of life.
METHODS: Data were utilised from the UK cohort of the PIVOT-vWD study (Oct 24 - Jan 25); a direct-to-community, cross-sectional study capturing outcomes of vWD across 116 individuals. Respondents reported patient and clinical characteristics, alongside the EQ-5D-5L and AQoL-4D (self and/or proxy-reported versions). Participants were grouped by their self-reported vWD type (Type 1, 2 and 3); those ‘unreported’ or ‘acquired’ were excluded. Distribution of responses across domains of each HRQoL instrument were assessed using frequency and percentage. Spearman’s rank coefficients were calculated to analyse associations between instrument domains.
RESULTS: The included sample totalled 104 individuals (86 self-complete/18 proxy-complete). EQ-5D-5L results were similar for Type 1 and 2, with 27% vs. 25% reporting moderate to extreme problems in mobility, 12% vs. 12% in self-care, and 30% vs. 32% in usual activities. These rose in Type 3 to 83%, 50%, and 67% respectively; pain/discomfort (27-29% vs. 43%) and anxiety/depression (18-20% vs. 43%) also increased. Across 12 AQoL-4D items, responses were similar for Types 1 and 2. Type 3 reported more problems with self-care, mobility, household tasks, family role, and pain. Sleep, anxiety, and friendships showed moderate burden across types; hearing and communication were rarely affected. Convergent correlations were strongest for anxiety (rs=0.87) and pain (rs=0.82). Sensory and communication domains were more distinct to AQoL-4D (rs=<0.33 across EQ-5D domains).
CONCLUSIONS: HRQoL burden was greatest in Type 3 vWD, while responses were broadly similar between Type 1 and 2 across both instruments. While both tools captured physical and emotional impacts, AQoL-4D may offer added value by capturing broader social and sensory domains relevant to quality of life.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR23
Topic
Methodological & Statistical Research, Patient-Centered Research, Study Approaches
Topic Subcategory
Instrument Development, Validation, & Translation, 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)