ArticleMuscle & nerve2026
Exploring the Content Validity of Patient-Reported Outcome Measures to Capture the Patient Experience of Becker Muscular Dystrophy.
Article in Muscle & nerve, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
Abstract
INTRODUCTION/
aimsThe patient experience of Becker muscular dystrophy (BMD) is not well understood, making it difficult to evaluate the conceptual relevance of proposed patient-reported outcome (PRO) measures. This study aimed to conceptualize the patient experience of BMD and evaluate content validity and perceptions of meaningful changes of PRO measures in this population.
methodsSemi-structured interviews were conducted with ambulatory adult and adolescent US-based participants with BMD. Transcripts were analyzed using content and framework analysis. Participant quotes were coded to identify concepts and themes, illustrated in a conceptual model of BMD experience. Cognitive debriefing methods assessed patient understanding of the brief pain inventory-short form (BPI-SF), quality of life in neurological disorders (Neuro-QoL), and three patient-reported outcomes measurement information system (PROMIS-57) domains: Physical Function, Fatigue, and Pain Interference.
resultsTwenty-nine patients (mean age: 27.0 years; mean age at BMD diagnosis: 15.1 years) and two caregivers described a wide range of signs, symptoms, and impacts. Physical fatigue, pain, weakness, and impacts on physical mobility and emotional wellbeing were considered most bothersome. The BPI-SF Item 3 and PROMIS-57 domains were well understood and relevant; however, the Neuro-QoL Upper Extremity questionnaire demonstrated low conceptual relevance. DISCUSSION: The conceptual model illustrated the burden of living with BMD. The BPI-SF Item 3 and evaluated PROMIS-57 domains are considered content-valid PRO measures in BMD, with most participants associating score improvements with meaningful changes to their health-related quality of life.
Indexed as
Identifiers
What Socratic holds
Registered trials
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 Socratic graph.