Health-related quality of life in Chinese boys with Duchenne muscular dystrophy and their families

Abstract

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 (<i>PedsQL</i> 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 <i>PedsQL</i> 4.0 Generic Core Scale scores for the boys with DMD were significantly lower than those of age-matched healthy boys, for overall (<i>p</i> < 0.05, parent-report; <i>p</i> <0.001, self-report), physical (<i>p</i> < 0.001, parent-report and self-report), and social (<i>p</i> < 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 <i>PedsQL</i> FIM total score showed an inverse relationship with the affected child's age (correlation coefficient: -0.55; <i>p</i> < 0.01) and the disease stage (correlation coefficient: -0.63; <i>p</i> < 0.01) confirming that parental HRQOL and overall family functioning worsened as the child increased in age with advancing disease stage.