Background: Although some studies have examined behavior and quality of life in Duchenne Muscular Dystrophy (DMD), nearly all focus on caregiver perceptions, and there are none that describe the patients’ perception of self with their personal quality of life.
Objectives: This study aimed to learn how patients view their own behavior and adaptive skills and how it impacts their quality of life in comparison with parent/caregiver perceptions.
Approach: 41 boys and men with DMD were recruited through the University of Virginia (UVA) Children’s Hospital Neuromuscular Clinic. Each patient and caregiver dyad completed a series of questionnaires, including the Behavior Assessment System for Children, 2nd edition (BASC-2), a multi-item inventory that addresses adaptive skills, behavioral symptoms, externalizing and internalizing problems, and school problems, and the Pediatric Quality of Life Inventory for DMD (PedsQL for DMD), which rates patient challenges and abilities in four domains of personal importance. This study was approved by the UVA IRB.
Results: Overall, boys and men with DMD rated most of their behavior and adaptive skills using the BASC-2 similarly to the general population, with only modest increases in the number scores in the concerning range for attitude to school, social stress, interpersonal relations, self-esteem, and self-reliance. Well over half of parent assessments, however, rated problems with social skills, leadership, activities of daily living, and functional communication. When compared with quality of life scores, worse BASC-2 scores for both self and parent assessments correlated with lower scores in the worry and communication PedsQL domains but not in the activity and treatment domains. Average quality of life scores reported by the patients were higher than those perceived by the parents in each domain except worry.
Conclusions: Boys and men with DMD rate their adaptive skills, behavioral symptoms, externalizing and internalizing problems, and school problems, more positively than parents/caregivers, providing evidence that self reports on behavior and adaptive challenges should always be attempted. More research is needed in order to determine if this difference is due to discrepancy in interpretation, comprehension, or expectations. In line with previous studies comparing patients with DMD and their parents’ perceptions of quality of life, parents underestimate their children’s quality of life in most domains