ArticleNeuromuscular disorders : NMD2016
Health-related quality of life and functional changes in DMD: A 12-month longitudinal cohort study.
Article in Neuromuscular disorders : NMD, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03127241 (USEFUL), which is not on this map. Cited by 21 papers, 2 of them syntheses that pooled it.
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.
USEFUL: User-centred Assistive SystEm for Arm Functions in neUromuscuLar Subjects
Who cites it
21 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Duchenne muscular dystrophy in Italy: A systematic review of epidemiology, quality of life, treatment adherence, and economic impact.PloS one · 2023Pooled it
- Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments.Health and quality of life outcomes · 2020Pooled it
- Exploring the relationship between North Star Ambulatory Assessment and Health Utilities Index scores in Duchenne muscular dystrophy.Health and quality of life outcomes · 2023Trial
- Living with Duchenne Muscular Dystrophy Beyond the Physical Implications: Cognitive Features, Psychopathology Aspects, and Psychosocial Resources-A Narrative Review.Brain sciences · 2025Review
- Socioeconomic determinants of the quality of life in boys suffering from Duchenne muscular dystrophy & their caregivers.The Indian journal of medical research · 2025Article
- "If you cannot measure it, you cannot improve it". Outcome measures in Duchenne Muscular Dystrophy: current and future perspectives.Acta neurologica Belgica · 2025Review
- Quality of life assessment instruments in children and adolescents with neuromuscular diseases: a systematic scoping review.Health and quality of life outcomes · 2024Article
- Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease.Journal of neuromuscular diseases · 2024Article
- Spinal muscular atrophy.Nature reviews. Disease primers · 2022Review
- Medical student acceptance on gene therapy to increase children's well-being with genetic diseases: a study in Indonesia.Future science OA · 2022Article
- Bone health in Duchenne muscular dystrophy: clinical and biochemical correlates.Journal of endocrinological investigation · 2022Observational
- Article
- The impact of testosterone therapy on quality of life in adolescents with Duchenne muscular dystrophy.Neuromuscular disorders : NMD · 2021Article
- Quality of Life in SMA Patients Under Treatment With Nusinersen.Frontiers in neurology · 2021Article
- "Be an ambassador for change that you would like to see": a call to action to all stakeholders for co-creation in healthcare and medical research to improve quality of life of people with a neuromuscular disease.Orphanet journal of rare diseases · 2019Article
- Incomplete description of the current body of evidence of the health economics of Duchenne muscular dystrophy.Orphanet journal of rare diseases · 2019Review
- Article
- A review of quality of life themes in Duchenne muscular dystrophy for patients and carers.Health and quality of life outcomes · 2018Review
- Measuring quality of life in children with spinal muscular atrophy: a systematic literature review.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2018Review
- The Italian neuromuscular registry: a coordinated platform where patient organizations and clinicians collaborate for data collection and multiple usage.Orphanet journal of rare diseases · 2018Article
Corrections and comments
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Authors and funding
26 authors.
Funding
Abstract
In Duchenne muscular dystrophy (DMD) little has been reported on the association between clinical outcome measures and patient health-related quality of life (HRQOL) tools. Our study evaluated the relationship between 12 month changes on the Generic Core Scales (GCS), the Multidimensional Fatigue Scale and the Neuromuscular Module of the PedsQL(TM) with several outcome measures (6 minute walk test, North Star Ambulatory Assessment and timed items) in ambulatory DMD. Ninety-eight ambulatory DMD in a multicentric setting were included in the study. At baseline, the PedsQL(TM) inventories correlated with almost all the functional measures On the Child Self-Report there was a significant decrease between baseline and 12 months on the PedsQL(TM) GCS and its first domain, in parallel with the decrement in the functional outcome measures. Correlation between the 12 month changes on the PedsQL(TM) inventories and functional measures were almost all negligible. Similar results were obtained on the Parent Proxy-Report. In conclusion, PedsQL(TM) correlates with the level of impairment at baseline, but this does not hold true when 12 month changes are considered. Further studies comparing different tools are needed to better elucidate the complexity of the relationship between HRQOL and functional performances.
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