Houwen-van Opstal SL, Jansen M, van Alfen N, de Groot IJ. Health-related quality of life and its relation to disease severity in boys with Duchenne muscular dystrophy: satisfied boys, worrying parents–a case-control study. Journal of child neurology 2014;29:1486-95
This study aims to assess the family functioning and health-related quality of life (HRQOL) in Chinese boys with Duchenne muscular dystrophy (DMD) and their parents using Pediatric Quality-of-Life Family Impact Module (PedsQL FIM) and Pediatric Quality-of-Life Inventory (PedsQL) 4.0. Findings from 15 families with DMD were compared with 15 unaffected families. The HRQOL, as measured by the mean PedsQL 4.0 Generic Core Scale scores for the boys with DMD were significantly lower than those of age-matched healthy boys, for overall (p < 0.05, parent-report; p <0.001, self-report), physical (p < 0.001, parent-report and self-report), and social (p < 0.05, parent-report) functioning, but the emotional functioning is not affected. The parent-child concordance of our affected DMD families was generally in the moderate-to-good agreement range (intraclass correlation coefficients from 0.51 to 0.73), except for emotional (0.28) and social (0.31) functioning. The PedsQL FIM total score showed an inverse relationship with the affected child’s age (correlation coefficient: -0.55; p < 0.01) and the disease stage (correlation coefficient: -0.63; p < 0.01) confirming that parental HRQOL and overall family functioning worsened as the child increased in age with advancing disease stage.