Health-related quality of life in children and adolescents with Duchenne muscular dystrophy

Karen Uzark, Eileen King, Linda Cripe, Robert Spicer, Jackie Sage, Kathleen Kinnett, Brenda Wong, Jesse Pratt, James W. Varni

Research output: Contribution to journalArticle

63 Citations (Scopus)

Abstract

OBJECTIVES: 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.

Original languageEnglish (US)
Pages (from-to)e1559-e1566
JournalPediatrics
Volume130
Issue number6
DOIs
StatePublished - Dec 2012

Fingerprint

Duchenne Muscular Dystrophy
Quality of Life
Parents
Self Report
Steroids
Boys
Health
Pediatrics
Equipment and Supplies

Keywords

  • Adolescents
  • Duchenne muscular dystrophy
  • PedsQL
  • Psychosocial
  • Quality of life

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

Cite this

Uzark, K., King, E., Cripe, L., Spicer, R., Sage, J., Kinnett, K., ... Varni, J. W. (2012). Health-related quality of life in children and adolescents with Duchenne muscular dystrophy. Pediatrics, 130(6), e1559-e1566. https://doi.org/10.1542/peds.2012-0858

Health-related quality of life in children and adolescents with Duchenne muscular dystrophy. / Uzark, Karen; King, Eileen; Cripe, Linda; Spicer, Robert; Sage, Jackie; Kinnett, Kathleen; Wong, Brenda; Pratt, Jesse; Varni, James W.

In: Pediatrics, Vol. 130, No. 6, 12.2012, p. e1559-e1566.

Research output: Contribution to journalArticle

Uzark, K, King, E, Cripe, L, Spicer, R, Sage, J, Kinnett, K, Wong, B, Pratt, J & Varni, JW 2012, 'Health-related quality of life in children and adolescents with Duchenne muscular dystrophy', Pediatrics, vol. 130, no. 6, pp. e1559-e1566. https://doi.org/10.1542/peds.2012-0858
Uzark, Karen ; King, Eileen ; Cripe, Linda ; Spicer, Robert ; Sage, Jackie ; Kinnett, Kathleen ; Wong, Brenda ; Pratt, Jesse ; Varni, James W. / Health-related quality of life in children and adolescents with Duchenne muscular dystrophy. In: Pediatrics. 2012 ; Vol. 130, No. 6. pp. e1559-e1566.
@article{c8032c6f1bcc4f5383f47f07e8e484a4,
title = "Health-related quality of life in children and adolescents with Duchenne muscular dystrophy",
abstract = "OBJECTIVES: 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.",
keywords = "Adolescents, Duchenne muscular dystrophy, PedsQL, Psychosocial, Quality of life",
author = "Karen Uzark and Eileen King and Linda Cripe and Robert Spicer and Jackie Sage and Kathleen Kinnett and Brenda Wong and Jesse Pratt and Varni, {James W.}",
year = "2012",
month = "12",
doi = "10.1542/peds.2012-0858",
language = "English (US)",
volume = "130",
pages = "e1559--e1566",
journal = "Pediatrics",
issn = "0031-4005",
publisher = "American Academy of Pediatrics",
number = "6",

}

TY - JOUR

T1 - Health-related quality of life in children and adolescents with Duchenne muscular dystrophy

AU - Uzark, Karen

AU - King, Eileen

AU - Cripe, Linda

AU - Spicer, Robert

AU - Sage, Jackie

AU - Kinnett, Kathleen

AU - Wong, Brenda

AU - Pratt, Jesse

AU - Varni, James W.

PY - 2012/12

Y1 - 2012/12

N2 - OBJECTIVES: 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.

AB - OBJECTIVES: 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.

KW - Adolescents

KW - Duchenne muscular dystrophy

KW - PedsQL

KW - Psychosocial

KW - Quality of life

UR - http://www.scopus.com/inward/record.url?scp=84870517916&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84870517916&partnerID=8YFLogxK

U2 - 10.1542/peds.2012-0858

DO - 10.1542/peds.2012-0858

M3 - Article

C2 - 23129083

AN - SCOPUS:84870517916

VL - 130

SP - e1559-e1566

JO - Pediatrics

JF - Pediatrics

SN - 0031-4005

IS - 6

ER -