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Updated: Jul 14, 2026
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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Estimating Utility Values Using the DMD-QoL: A Disease-Specific Preference-Based Measure for Duchenne Muscular
Andrea Bever1, Shelagh M Szabo2, Pramoda Jayasinghe1
1Broadstreet Health Economics & Outcomes Research, 300 - 177 West 7th Ave, Vancouver, BC, V5Y 1L8, Canada.
Advances in Therapy
|July 13, 2026
Summary
The DMD-QoL utility measure remained stable in Duchenne muscular dystrophy (DMD) patients over 12 months. Unlike the Health Utilities Index (HUI-2), DMD-QoL did not show declines with worsening mobility, reflecting its broader quality of life conceptualization.
Area of Science:
- Health Economics and Outcomes Research
- Clinical Trial Methodology
- Patient-Reported Outcomes
Background:
- The Duchenne Muscular Dystrophy Quality of Life (DMD-QoL) is a preference-based measure (PBM) designed for individuals with Duchenne muscular dystrophy (DMD).
- It aims to capture a broader concept of wellness and quality of life (QoL) compared to existing PBMs.
- This study sought to estimate and compare the utility values derived from DMD-QoL and the Health Utilities Index (HUI-2) in DMD patients.
Purpose of the Study:
- To estimate and compare utility values derived from the DMD-QoL and HUI-2 in individuals with Duchenne muscular dystrophy (DMD).
- To assess the responsiveness of both measures to changes in health status, particularly ambulatory function, over a 12-month period.
- To evaluate the conceptual alignment of DMD-QoL with the lived experiences of individuals with DMD.
Main Methods:
- 155 individuals with DMD or their caregivers completed the DMD-QoL and HUI-2 at baseline and 12 months.
- Participants were categorized into health states based on upper and lower limb function.
- Mean utility values and changes over 12 months were calculated by health state and respondent type, including analysis of utility changes in participants experiencing declining ambulatory function.
Main Results:
- Mean baseline DMD-QoL utility ranged from 0.62 to 0.78 (patient-assessed) and 0.63 to 0.84 (caregiver-assessed) across health states.
- Mean baseline HUI-2 values ranged from 0.49 to 0.96 (patient-assessed) and 0.44 to 0.85 (caregiver-assessed).
- While both measures were stable over 12 months, HUI-2 showed a significant decline (-0.16) in participants with worsening ambulatory function, whereas DMD-QoL utility remained stable (0.00).
Conclusions:
- DMD-QoL utilities showed less variation across health states compared to HUI-2 scores.
- HUI-2 scores demonstrated sensitivity to declining mobility, a change not reflected in DMD-QoL scores.
- The stability of DMD-QoL scores suggests its broader conceptualization of QoL, grounded in the experiences of individuals with DMD, may be more appropriate for this population.
