The PedsQL™ in pediatric patients with duchenne muscular dystrophy: Feasibility, reliability, and validity of the pediatric quality of life inventory neuromuscular module and generic core scales

Sarah E. Davis, Linda S. Hynan, Christine A. Limbers, C. Mariam Andersen, Medrith C. Greene, James W. Varni, Susan T. Iannaccone

Research output: Contribution to journalArticle

64 Citations (Scopus)

Abstract

Objective: To evaluate the reliability and validity of the PedsQL™ 3.0 Neuromuscular Module (NMM) in assessing health-related quality of life in the Duchenne muscular dystrophy (DMD) population for use as a secondary outcome measure in phase III clinical trials. Background: DMD is the most common genetic form of muscular dystrophy in childhood. Clinical trials are underway to evaluate modalities of treatment. The NMM was developed based on interviews of patients with DMD and spinal muscular atrophy. To determine the PedsQL™ reliability and validity, we administered the NMM to patients with DMD and their caregivers. Design/Methods: Boys 8 to 18 years old with DMD were recruited from a neuromuscular disease clinic. At baseline, the child and caregiver completed the NMM and the PedsQL™ 4.0 Generic Core Scales (GC). The NMM was repeated 2 to 6 weeks later. Reliability was assessed using Cronbach's coefficient alpha (internal consistency) and intraclass correlation (ICC) (test-retest consistency). Construct validity was assessed by comparing baseline child and caregiver NMM total scores with the GC Total Score, forced vital capacity, cardiac ejection fraction, and ambulatory status. Results: Forty-four children and their caregivers completed the study. Internal consistency reliability of the total scale score of the NMM was demonstrated (Child α = 0.85; Caregiver α = 0.87). Test-retest reliability of the NMM was also demonstrated (Child ICC = 0.75, P = 0.001; Caregiver ICC = 0.85, P < 0.001). Validity of the total scale score of the NMM when compared with the GC Total Scale Score was supported (Child r (41) = 0.63, P < 0.001; Caregiver r (42) = 0.64, P < 0.001). Validity of the NMM compared with forced vital capacity was also supported (Child r (38) = 0.35, P = 0.032; Caregiver r (39) = 0.41, P = 0.01). The NMM parent-proxy-report and child self-report "About My Child's Neuromuscular Disease" scale was significantly related to wheelchair use (P < 0.008 and 0.016, respectively); the GC "Child Self-Report "Physical Health" scale was also significantly related to wheelchair use (P < 0.001). We were unable to conduct any analysis with ejection fraction because of the small number of children across all categories. Conclusions: The PedsQL™ NMM is a reliable measure of disease-specific health-related quality of life in the DMD population and may be used as an outcome measure in clinical trials.

Original languageEnglish (US)
Pages (from-to)97-109
Number of pages13
JournalJournal of Clinical Neuromuscular Disease
Volume11
Issue number3
DOIs
StatePublished - Mar 2010

Fingerprint

Duchenne Muscular Dystrophy
Reproducibility of Results
Quality of Life
Pediatrics
Caregivers
Equipment and Supplies
Neuromuscular Diseases
Wheelchairs
Vital Capacity
Self Report
Outcome Assessment (Health Care)
Clinical Trials
Spinal Muscular Atrophy
Phase III Clinical Trials
Muscular Dystrophies
Proxy
Population
Interviews

Keywords

  • Duchenne muscular dystrophy
  • Health related quality of life
  • Neuromuscular Module
  • Pediatric quality of life
  • PedsQL™

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology

Cite this

The PedsQL™ in pediatric patients with duchenne muscular dystrophy : Feasibility, reliability, and validity of the pediatric quality of life inventory neuromuscular module and generic core scales. / Davis, Sarah E.; Hynan, Linda S.; Limbers, Christine A.; Andersen, C. Mariam; Greene, Medrith C.; Varni, James W.; Iannaccone, Susan T.

In: Journal of Clinical Neuromuscular Disease, Vol. 11, No. 3, 03.2010, p. 97-109.

Research output: Contribution to journalArticle

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abstract = "Objective: To evaluate the reliability and validity of the PedsQL™ 3.0 Neuromuscular Module (NMM) in assessing health-related quality of life in the Duchenne muscular dystrophy (DMD) population for use as a secondary outcome measure in phase III clinical trials. Background: DMD is the most common genetic form of muscular dystrophy in childhood. Clinical trials are underway to evaluate modalities of treatment. The NMM was developed based on interviews of patients with DMD and spinal muscular atrophy. To determine the PedsQL™ reliability and validity, we administered the NMM to patients with DMD and their caregivers. Design/Methods: Boys 8 to 18 years old with DMD were recruited from a neuromuscular disease clinic. At baseline, the child and caregiver completed the NMM and the PedsQL™ 4.0 Generic Core Scales (GC). The NMM was repeated 2 to 6 weeks later. Reliability was assessed using Cronbach's coefficient alpha (internal consistency) and intraclass correlation (ICC) (test-retest consistency). Construct validity was assessed by comparing baseline child and caregiver NMM total scores with the GC Total Score, forced vital capacity, cardiac ejection fraction, and ambulatory status. Results: Forty-four children and their caregivers completed the study. Internal consistency reliability of the total scale score of the NMM was demonstrated (Child α = 0.85; Caregiver α = 0.87). Test-retest reliability of the NMM was also demonstrated (Child ICC = 0.75, P = 0.001; Caregiver ICC = 0.85, P < 0.001). Validity of the total scale score of the NMM when compared with the GC Total Scale Score was supported (Child r (41) = 0.63, P < 0.001; Caregiver r (42) = 0.64, P < 0.001). Validity of the NMM compared with forced vital capacity was also supported (Child r (38) = 0.35, P = 0.032; Caregiver r (39) = 0.41, P = 0.01). The NMM parent-proxy-report and child self-report {"}About My Child's Neuromuscular Disease{"} scale was significantly related to wheelchair use (P < 0.008 and 0.016, respectively); the GC {"}Child Self-Report {"}Physical Health{"} scale was also significantly related to wheelchair use (P < 0.001). We were unable to conduct any analysis with ejection fraction because of the small number of children across all categories. Conclusions: The PedsQL™ NMM is a reliable measure of disease-specific health-related quality of life in the DMD population and may be used as an outcome measure in clinical trials.",
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T2 - Feasibility, reliability, and validity of the pediatric quality of life inventory neuromuscular module and generic core scales

AU - Davis, Sarah E.

AU - Hynan, Linda S.

AU - Limbers, Christine A.

AU - Andersen, C. Mariam

AU - Greene, Medrith C.

AU - Varni, James W.

AU - Iannaccone, Susan T.

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N2 - Objective: To evaluate the reliability and validity of the PedsQL™ 3.0 Neuromuscular Module (NMM) in assessing health-related quality of life in the Duchenne muscular dystrophy (DMD) population for use as a secondary outcome measure in phase III clinical trials. Background: DMD is the most common genetic form of muscular dystrophy in childhood. Clinical trials are underway to evaluate modalities of treatment. The NMM was developed based on interviews of patients with DMD and spinal muscular atrophy. To determine the PedsQL™ reliability and validity, we administered the NMM to patients with DMD and their caregivers. Design/Methods: Boys 8 to 18 years old with DMD were recruited from a neuromuscular disease clinic. At baseline, the child and caregiver completed the NMM and the PedsQL™ 4.0 Generic Core Scales (GC). The NMM was repeated 2 to 6 weeks later. Reliability was assessed using Cronbach's coefficient alpha (internal consistency) and intraclass correlation (ICC) (test-retest consistency). Construct validity was assessed by comparing baseline child and caregiver NMM total scores with the GC Total Score, forced vital capacity, cardiac ejection fraction, and ambulatory status. Results: Forty-four children and their caregivers completed the study. Internal consistency reliability of the total scale score of the NMM was demonstrated (Child α = 0.85; Caregiver α = 0.87). Test-retest reliability of the NMM was also demonstrated (Child ICC = 0.75, P = 0.001; Caregiver ICC = 0.85, P < 0.001). Validity of the total scale score of the NMM when compared with the GC Total Scale Score was supported (Child r (41) = 0.63, P < 0.001; Caregiver r (42) = 0.64, P < 0.001). Validity of the NMM compared with forced vital capacity was also supported (Child r (38) = 0.35, P = 0.032; Caregiver r (39) = 0.41, P = 0.01). The NMM parent-proxy-report and child self-report "About My Child's Neuromuscular Disease" scale was significantly related to wheelchair use (P < 0.008 and 0.016, respectively); the GC "Child Self-Report "Physical Health" scale was also significantly related to wheelchair use (P < 0.001). We were unable to conduct any analysis with ejection fraction because of the small number of children across all categories. Conclusions: The PedsQL™ NMM is a reliable measure of disease-specific health-related quality of life in the DMD population and may be used as an outcome measure in clinical trials.

AB - Objective: To evaluate the reliability and validity of the PedsQL™ 3.0 Neuromuscular Module (NMM) in assessing health-related quality of life in the Duchenne muscular dystrophy (DMD) population for use as a secondary outcome measure in phase III clinical trials. Background: DMD is the most common genetic form of muscular dystrophy in childhood. Clinical trials are underway to evaluate modalities of treatment. The NMM was developed based on interviews of patients with DMD and spinal muscular atrophy. To determine the PedsQL™ reliability and validity, we administered the NMM to patients with DMD and their caregivers. Design/Methods: Boys 8 to 18 years old with DMD were recruited from a neuromuscular disease clinic. At baseline, the child and caregiver completed the NMM and the PedsQL™ 4.0 Generic Core Scales (GC). The NMM was repeated 2 to 6 weeks later. Reliability was assessed using Cronbach's coefficient alpha (internal consistency) and intraclass correlation (ICC) (test-retest consistency). Construct validity was assessed by comparing baseline child and caregiver NMM total scores with the GC Total Score, forced vital capacity, cardiac ejection fraction, and ambulatory status. Results: Forty-four children and their caregivers completed the study. Internal consistency reliability of the total scale score of the NMM was demonstrated (Child α = 0.85; Caregiver α = 0.87). Test-retest reliability of the NMM was also demonstrated (Child ICC = 0.75, P = 0.001; Caregiver ICC = 0.85, P < 0.001). Validity of the total scale score of the NMM when compared with the GC Total Scale Score was supported (Child r (41) = 0.63, P < 0.001; Caregiver r (42) = 0.64, P < 0.001). Validity of the NMM compared with forced vital capacity was also supported (Child r (38) = 0.35, P = 0.032; Caregiver r (39) = 0.41, P = 0.01). The NMM parent-proxy-report and child self-report "About My Child's Neuromuscular Disease" scale was significantly related to wheelchair use (P < 0.008 and 0.016, respectively); the GC "Child Self-Report "Physical Health" scale was also significantly related to wheelchair use (P < 0.001). We were unable to conduct any analysis with ejection fraction because of the small number of children across all categories. Conclusions: The PedsQL™ NMM is a reliable measure of disease-specific health-related quality of life in the DMD population and may be used as an outcome measure in clinical trials.

KW - Duchenne muscular dystrophy

KW - Health related quality of life

KW - Neuromuscular Module

KW - Pediatric quality of life

KW - PedsQL™

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