ArticleJournal of child neurology2010
Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy.
Article in Journal of child neurology, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00312247 (Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy), which is not on this map. Cited by 27 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy
Who cites it
27 citing papers in PubMed, 2 syntheses or guidelines pooled 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
- The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review.Orphanet journal of rare diseases · 2017Pooled it
- Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.JAMA network open · 2022Trial
- Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.Annals of neurology · 2016Trial
- The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.Muscle & nerve · 2013Trial
- Living with Duchenne Muscular Dystrophy Beyond the Physical Implications: Cognitive Features, Psychopathology Aspects, and Psychosocial Resources-A Narrative Review.Brain sciences · 2025Review
- "If you cannot measure it, you cannot improve it". Outcome measures in Duchenne Muscular Dystrophy: current and future perspectives.Acta neurologica Belgica · 2025Review
- Reliability and construct validity of the Duchenne Video Assessment.Muscle & nerve · 2021Article
- Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy.Molecular genetics & genomic medicine · 2021Article
- The Minimal Clinical Important Difference (MCID) in Annual Rate of Change of Timed Function Tests in Boys with DMD.Journal of neuromuscular diseases · 2021Article
- Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study.Journal of comparative effectiveness research · 2020Article
- Quality of life in adults with muscular dystrophy.Health and quality of life outcomes · 2019Article
- The Lifelong Impact of X-Linked Hypophosphatemia: Results From a Burden of Disease Survey.Journal of the Endocrine Society · 2019Article
- Incomplete description of the current body of evidence of the health economics of Duchenne muscular dystrophy.Orphanet journal of rare diseases · 2019Review
- 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
- Characteristics of pain, other symptoms and function in pediatric post-thrombotic syndrome.Research and practice in thrombosis and haemostasis · 2018Article
- Mitochondrial disease patient motivations and barriers to participate in clinical trials.PloS one · 2018Article
- Health-related quality of life in patients with Duchenne muscular dystrophy: a multinational, cross-sectional study.Developmental medicine and child neurology · 2016Observational
- Health-related quality of life and functional changes in DMD: A 12-month longitudinal cohort study.Neuromuscular disorders : NMD · 2016Article
- Health-Related Quality of Life in Children with Duchenne Muscular Dystrophy: A Review.Journal of neuromuscular diseases · 2015Article
Corrections and comments
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Authors and funding
8 authors.
Funding
Abstract
In Duchenne muscular dystrophy, data directly linking changes in clinical outcome measures to patient-perceived well-being are lacking. This study evaluated the relationship between clinical outcome measures used in clinical trials of ambulatory Duchenne muscular dystrophy (Vignos functional grade, quantitative knee extension strength, timed functional performance measures, and gait velocity) and 2 health-related quality of life measures--the Pediatric Outcomes Data Collection Instrument and Pediatric Quality of Life Inventory-in 52 ambulatory Duchenne muscular dystrophy subjects and 36 controls. Those with the disease showed significant decrements in parent proxy-reported health-related quality of life measures versus controls across all domains. The Pediatric Outcomes Data Collection Instrument transfers/basic mobility and sports/ physical function and the Pediatric Quality of Life Inventory physical functioning domains had significant associations with age (and hence disease progression) and traditional clinical outcome measures employed in clinical trials of ambulatory boys with Duchenne muscular dystrophy. Selected domains of the Pediatric Outcomes Data Collection Instrument and generic Pediatric Quality of Life Inventory are potential patient-reported outcome measures for clinical trials in ambulatory individuals with the disease.
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Registered trials
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