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Hess, Robyn S.

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This cross-sectional study examined the influence that age/disease severity, pain, and/or family functioning have on different domains of health-related quality of life (HRQOL) from the perspectives of patients with a degenerative neuromuscular disease and their parents. Participants included 44 parents/caregivers and 39 patients between the ages of 6- and 25-years, who have a confirmed genetic diagnosis of Becker Muscular Dystrophy (BMD) or Duchene Muscular Dystrophy (DMD). Patients and/or their caregivers completed measures of HRQOL (PedsQLTM 4.0 Generic Core scales, Varni et al., 1999) and family functioning and well-being (PedsQLTM 2.0 Family Impact Module, Varni et al., 2004), as well as provided general child and family demographic information. Details regarding patients’ disease status (i.e., usage of steroids and respiratory-related devices, secondary medical conditions, and comorbid psychiatric and neurodevelopmental disorders) were also obtained from their medical charts. Univariate analyses were used to explore characteristics of the sample, disease-specific variables, and all survey data. Overall, patients and their caregivers reported low levels of physical and psychosocial HRQOL. Mean scores of caregiver well-being and family functioning were lower for this sample than for other studies using the same measure with families of pediatric populations. Furthermore, a series of multivariable regression analyses were conducted to assess associations between potential correlates and HRQOL scores. Age, family functioning, and/or pain accounted for a significant amount of the variance in child-reported and parent-reported Psychosocial Health Summary scores. The child-reported Physical Health Summary score also demonstrated a statistically significant model, with age and family functioning having a large influence. These results suggest that assessing and addressing concerns regarding pain and family functioning may potentially enhance HRQOL in patients with DMD.


150 pages

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