ArticleAdvances in therapy2026
Estimating Utility Values Using the DMD-QoL: A Disease-Specific Preference-Based Measure for Duchenne Muscular Dystrophy (DMD).
Article in Advances in therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
1 citing paper in PubMed.
- Systematic Literature Reviews of Health-State Utility Values, Costs, and Healthcare Resource Use in Duchenne Muscular Dystrophy.Journal of health economics and outcomes research · 2026Article
Corrections and comments
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Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionThe DMD-QoL is a disease-specific, preference-based measure (PBM). Its design was informed by the experiences of individuals with Duchenne muscular dystrophy (DMD), and it was developed to reflect a broader conceptualization of wellness and quality of life (QoL) compared with other PBMs. This study estimated and compared DMD-QoL and Health Utilities Index (HUI) utility in DMD.
methodsIndividuals with DMD or their caregivers completed the DMD-QoL and HUI-2 at baseline and 12 months. Participants were classified into health states based on upper and lower limb function. Mean utility and change over 12 months were calculated by baseline health state and respondent type; change in utility was also estimated among those in whom declining ambulatory function led to a health state transition.
resultsAmong 155 participants (caregiver-respondents, 78.7%), mean (standard deviation [SD]) patient age at baseline was 14.1 (6.9) years. Across health states, mean (SD) baseline DMD-QoL utility ranged from 0.62 (0.35) to 0.78 (0.10) (patient-assessed) and 0.63 (0.17) to 0.84 (0.12) (caregiver-assessed). Mean HUI-2 values ranged from 0.49 (0.07) to 0.96 (0.06) (patient-assessed) and 0.44 (0.09) to 0.85 (0.11) (caregiver-assessed). While DMD-QoL and HUI-2 utilities were relatively stable over 12 months, a clinically important and significant loss in HUI-2 utility (mean, - 0.16 [95% confidence interval, - 0.22, - 0.10]) was observed among 17 (11.0%) participants with worsening ambulatory function. No such change was observed with the DMD-QoL (0.00 [- 0.06, 0.06]).
conclusionDMD-QoL utilities did not vary meaningfully across health states compared with HUI-2 scores, which tended to decline with deteriorating mobility. The relative stability of DMD-QoL scores may reflect its broader conceptualization of QoL, grounded in the experiences of individuals with DMD.
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Registered trials
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