Self- and Caregiver-Reported Participation, Quality of Life, and Related Mood and Behavior Challenges in People Living With Dystrophinopathies

Abstract

BackgroundPrevious studies of quality of life (QOL), mood, and behavior in muscular dystrophy focus on caregiver perceptions. This cross-sectional study aims to determine the prevalence of clinically significant mood and behavior problems by both patient and caregiver report and assess relationship between mood/behavior and QOL. MethodsForty-one patients with dystrophinopathies (Duchenne muscular dystrophy [DMD] and Becker muscular dystrophy [BMD]) were recruited through the University of Virginia Neuromuscular Clinic. Each patient and caregiver dyad completed questionnaires, including the Behavior Assessment System for Children, 2nd Edition (BASC-2); the Pediatric Quality of Life Inventory for DMD (PedsQL-DMD); Children's Depression Inventory, 2nd Edition; and Screen for Child Anxiety Related Disorders. ResultsPersons with dystrophinopathies rated most of their behavior and adaptive skills similarly to the general population. Sixty-four percent of parent assessments rated clinically significant problems on the BASC-2. Worse BASC-2 scores for self- and parent assessments correlated with lower (worse) scores in the Worry and Communication PedsQL domains. Patient-reported QOL scores were higher than parent-reported scores in each domain except Worry. ConclusionsIndividuals with DMD/BMD rate their adaptive skills, behavioral symptoms, externalizing and internalizing problems, and school problems more positively than parents/caregivers. Obtaining self-report data is a worthwhile endeavor that can add value to intervention planning, with the ultimate goal of optimizing QOL.