Exploring the Content Validity of Patient-Reported Outcome Measures to Capture the Patient Experience of Becker Muscular Dystrophy.

Abstract

Introduction/aims: The 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.

Methods: Semi-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.

Results: Twenty-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.