EQ-5D Utility According to Patient Self-Reported Health on the Visual Analogue Scale (VAS) in Duchenne Muscular Dystrophy (DMD)
Author(s)
Filipovic Audhya I1, Szabo S2, Griffin EC2, Feeny D3, Malone D4, Neumann P5, Iannaccone ST6, Jayasinghe P2, Gooch KL1
1Sarepta Therapeutics, Inc., Cambridge, MA, USA, 2Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada, 3McMaster University, Hamilton, ON, Canada, 4University of Utah College of Pharmacy, Salt Lake City, UT, USA, 5Tufts University School of Medicine, Boston, MA, USA, 6The University of Texas Southwestern, Dallas, TX, USA
Presentation Documents
OBJECTIVES: The EQ-5D utility assessment considers general public perspective on health-related quality-of-life (HRQoL). In contrast, the EQ-5D VAS provides a respondent-focused assessment of self-rated health. The relationship between these measures in DMD is unclear. Therefore, this study examined correlations between EQ-5D utility and VAS scores, and variability in VAS score and utility values in DMD.
METHODS: EQ-5D and VAS responses from a sample of patients with DMD aged 12 to <40 years were analyzed. Patients were classified into four DMD-related health states by level of lower and upper limb function. Median (interquartile range, IQR) EQ-5D utility and VAS scores were compared based on health state by Kruskal-Wallis tests. Pearson’s correlation was used to assess the relationship between EQ-5D utility values and VAS scores. The range of utility scores per VAS score quartile was evaluated overall and stratified by patients’ ambulatory status.
RESULTS: The mean (SD) age of the 63 patients was 19.8 (6.1) years and 44 (69.8%) were non-ambulatory. Median (IQR) VAS scores were higher than utility values for the same respondents/health states. For example, for ‘early ambulatory’ boys (n=11) median (IQR) VAS score was 91 (85-96) and utility was 0.88 (0.69-0.92). Differences were greater for more severe health states; for ‘late non-ambulatory’ boys (n=23), median (IQR) VAS score was 87 (77.5-94.0) and utility was 0.26 (0.16-0.31). While median utility scores differed significantly by ambulatory status, median VAS scores did not. The correlation between VAS and utility scores was r=0.37 (p=0.003); r=0.81 (p<0.001) for ambulatory and r=0.36 (p=0.017) for non-ambulatory patients. Ranges of utility per VAS quartile showed large variability. For example, individuals in the best VAS score quartile (95-100) could have utility values between 0.26 and 1.00.
CONCLUSIONS: In DMD, the EQ-5D utility and VAS provide markedly different ratings of patient HRQoL, with divergences being greater with declining lower and upper limb function.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 12, S2 (December 2024)
Code
PCR29
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases