ArticleNeurology2021
Development of a New Quality of Life Measure for Duchenne Muscular Dystrophy Using Mixed Methods: The DMD-QoL.
Article in Neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers, 1 of them a synthesis that pooled it.
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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.
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Who cites it
35 citing papers in PubMed, 1 synthesis or guideline pooled it, 45 citations in OpenAlex.
- Measuring carer quality of life in Duchenne muscular dystrophy: a systematic review of the reliability and validity of self-report instruments using COSMIN.Health and quality of life outcomes · 2022Pooled 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
- Factors associated with the health-related quality of life among people with Duchenne muscular dystrophy: a study using the Health Utilities Index (HUI).Health and quality of life outcomes · 2022Trial
- Trial
- Estimating Utility Values Using the DMD-QoL: A Disease-Specific Preference-Based Measure for Duchenne Muscular Dystrophy (DMD).Advances in therapy · 2026Article
- The Impact of Clinical Trial Participation on Quality of Life in Duchenne Muscular Dystrophy.Pharmaceutical medicine · 2026Article
- Development and Validation of the Idiopathic Multicentric Castleman Disease Symptom Burden Scale (ISBUS): Protocol for an International Multistage Mixed Methods Study.JMIR research protocols · 2026Article
- Measuring health-related quality of life in infants and toddlers: conceptual challenges and proposed recommendations.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2026Article
- Development of a cost-effectiveness analysis model for Duchenne muscular dystrophy utilizing the national registry in Japan.Scientific reports · 2025Article
- Psychometric evaluation of the PROMIS parent proxy mobility item bank for use in Duchenne muscular dystrophy.Developmental medicine and child neurology · 2025Article
- Capturing what matters: Patient-reported LGI1-ANTibody encephalitis outcome RatiNg scale (LANTERN).Annals of clinical and translational neurology · 2025Article
- Patients' Experiences of an Active Transcutaneous Implant: The Bone Conduction Implant.Audiology & neuro-otology · 2025Article
- Burden of Duchenne muscular dystrophy in Australia: a scoping review.BMJ neurology open · 2025Article
- Psychometric analysis of the patient-reported outcomes measurement information system parent proxy physical function-upper extremity item bank for children with Duchenne muscular dystrophy.Frontiers in neurology · 2025Article
- The PompeQoL questionnaire: Development and validation of a new measure for children and adolescents with Pompe disease.Journal of inherited metabolic disease · 2024Article
- Living with Pompe disease: results from a qualitative interview study with children and adolescents and their caregivers.Orphanet journal of rare diseases · 2024Article
- Development of a novel patient reported outcome measure for health-related quality of life in amyotrophic lateral sclerosis (PROQuALS): study protocol.Health and quality of life outcomes · 2024Article
- Article
- "You Take This Day by Day, Come What May": A Qualitative Study of the Psychosocial Impacts of Living with Duchenne Muscular Dystrophy.Advances in therapy · 2024Article
- A scoping review to create a framework for the steps in developing condition-specific preference-based instruments de novo or from an existing non-preference-based instrument: use of item response theory or Rasch analysis.Health and quality of life outcomes · 2024Article
Corrections and comments
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Authors and funding
6 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveBased on concerns about existing patient-reported outcome measures (PROMs) for assessing quality of life (QoL) in Duchenne muscular dystrophy (DMD), we describe the mixed methods development of a new QoL PROM for use in boys and men with DMD: the DMD-QoL.
methodsThe DMD-QoL was developed in 3 stages. First, draft items were generated from 18 semistructured qualitative interviews with boys and men with DMD, analyzed using framework analysis. Second, cognitive debriefing interviews with patients (n = 10), clinicians (n = 8), and patients' parents (n = 10) were undertaken, and a reduced item set was selected and refined. Third, psychometric data on the draft items from a cross-sectional online survey (n = 102) and stakeholder input from patients and patients' parents were used to produce the final questionnaire. Patient and public involvement and engagement was embedded throughout the process.
resultsFrom an initial draft of 47 items, a revised set of 27 items was produced at stage 2, and this set was further refined at stage 3 to generate the DMD-QoL, a 14-item QoL PROM. The DMD-QoL is designed for use from 7 years of age by proxy report and from 10 years of age by self-report or proxy report. The final measure showed good psychometric properties.
conclusionThe DMD-QoL is a new 14-item QoL PROM for boys and men with DMD, with demonstrable content and face validity.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.