title: Health-related quality of life in children and adolescents with Duchenne muscular dystrophy.
abstract: OBJECTIVE
  The purpose of this study was to assess health-related quality of life (QoL) in children with Duchenne muscular dystrophy (DMD), including development and field-testing of a DMD-specific module integrated with the core Pediatric Quality of Life Inventory (PedsQL).
  
  
  METHODS
  The PedsQL 4.0 Generic Core and DMD Module Scales were completed by 203 families, including 200 parents and 117 boys with DMD. Scores on the PedsQL Core Scales were compared with those of matched healthy children. Relationships between PedsQL scores and patient characteristics were examined.
  
  
  RESULTS
  By both parent report and child self-report, mean PedsQL scores for boys with DMD were significantly lower than those for healthy children for physical and psychosocial QoL (P < .0001), with significantly impaired psychosocial QoL scores self-reported by 57%. Psychosocial QoL, by self-report only, tended to be higher in the older boys (13-18 years) than in younger boys (8-12 years; P = .05) and was not significantly associated with use of mobility aids. Although parents reported higher Daily Activities scores in boys receiving steroids (P = .01), boys receiving steroids reported no difference in Daily Activities but significantly less worry (P = .004). Parent-child concordance was generally in the fair to poor range. Internal consistency reliability coefficients for PedsQL DMD module scales ranged from 0.66 to 0.86.
  
  
  CONCLUSIONS
  Overall, boys with DMD reported significantly lower QoL than their healthy peers. Despite decreased physical functioning, older boys seem to perceive better psychosocial QoL than perceived by their parents and by younger boys, unrelated to their need for mobility aids.
authors.affiliation: Departments of Pediatric Cardiology, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio. karenu@med.umich.edu
authors.forename: Karen
authors.initials: K
authors.lastname: Uzark
authors.forename: Eileen
authors.initials: E
authors.lastname: King
authors.forename: Linda
authors.initials: L
authors.lastname: Cripe
authors.forename: Robert
authors.initials: R
authors.lastname: Spicer
authors.forename: Jackie
authors.initials: J
authors.lastname: Sage
authors.forename: Kathleen
authors.initials: K
authors.lastname: Kinnett
authors.forename: Brenda
authors.initials: B
authors.lastname: Wong
authors.forename: Jesse
authors.initials: J
authors.lastname: Pratt
authors.forename: James W
authors.initials: JW
authors.lastname: Varni
mesh_terms: D000203:Activities of Daily Living / Q000145:classification / Q000523:psychology; D000293:Adolescent; D000367:Age Factors; D000893:Anti-Inflammatory Agents / Q000008:administration & dosage / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D004185:Disability Evaluation; D018450:Disease Progression; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000523:psychology* / Q000534:rehabilitation; D020380:Needs Assessment; D011241:Prednisone / Q000008:administration & dosage / Q000009:adverse effects; D011282:Pregnenediones / Q000008:administration & dosage / Q000009:adverse effects; D011788:Quality of Life / Q000523:psychology*; D012016:Reference Values; D012803:Sick Role; D012917:Social Adjustment; D011795:Surveys and Questionnaires; D014910:Wheelchairs / Q000523:psychology
chemical_list: D000893:Anti-Inflammatory Agents; D011282:Pregnenediones; C021988:deflazacort; D011241:Prednisone