Trial reportHealth and quality of life outcomes2022
Factors associated with the health-related quality of life among people with Duchenne muscular dystrophy: a study using the Health Utilities Index (HUI).
Trial report in Health and quality of life outcomes, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01254019 (A Phase III, Randomized, Double Blind, Placebo-controlled Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy), which is not on this map. Cited by 8 papers.
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.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase III, Randomized, Double Blind, Placebo-controlled Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
Who cites it
8 citing papers in PubMed.
- Exploring the relationship between North Star Ambulatory Assessment and Health Utilities Index scores in Duchenne muscular dystrophy.Health and quality of life outcomes · 2023Trial
- Estimating Utility Values Using the DMD-QoL: A Disease-Specific Preference-Based Measure for Duchenne Muscular Dystrophy (DMD).Advances in therapy · 2026Article
- Economic Burden of Congenital Cytomegalovirus Disease on Families in the United States.Viruses · 2026Article
- Assessing the Relationship of Quality of Life With Functional Status in a Large Cohort of Adult Patients With Neuromuscular Disorders.Neurology. Clinical practice · 2026Article
- Assessing the socio-economic burden of inherited and inflammatory neuromuscular diseases (BIND study): a study protocol.Orphanet journal of rare diseases · 2025Article
- Household costs in the United States for accommodating functional impairments associated with Duchenne muscular dystrophy: results from a caregiver survey.Orphanet journal of rare diseases · 2025Article
- Socioeconomic determinants of the quality of life in boys suffering from Duchenne muscular dystrophy & their caregivers.The Indian journal of medical research · 2025Article
- Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L.Journal of patient-reported outcomes · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundData on health state utility in Duchenne muscular dystrophy (DMD) are few. This study estimated mean utility values by age, ambulatory status and over time, and investigated which aspects of health-related quality-of-life (HRQoL) are most strongly associated with utility in DMD.
methodsData from placebo-treated ambulant boys with DMD with exon 51 skip amenable mutations, (NCT01254019), were included. Ambulatory function assessments were conducted at baseline and every 12 weeks for the trial duration. Family member proxies completed the Health Utility Index (HUI) at baseline, 24 and 48 weeks; and HUI3 and HUI2 utility values were summarized. Changes in HUI attribute level over time, and predictors of changes in utility, were explored.
resultsSixty-one boys (mean [range] age of 8.0 [5-16] years) were included in the analysis. Mean baseline utilities were 0.82 (HUI3) and 0.87 (HUI2); and utilities were 0.35 (HUI3) and 0.55 (HUI2) after loss of ambulation (LOA, where applicable). Over the follow-up period mean utility declined more among the older versus younger boys. Pain accounted for the highest proportion of variability (42%) in change in HUI3 utility from baseline to week 48, while for HUI2, self-care (39%) did. After LOA, change in ambulation levels explained 88% of the decline in mean HUI3 utility and change in mobility levels explained 66% of the decline in mean HUI2 utility.
conclusionsUtility values among this sample were higher than previously published estimates. In younger boys utility remained relatively stable, but older boys and those losing ambulation experienced important declines over follow-up.
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