Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy

Craig M McDonald, Dawn A. McDonald, Anita Bagley, Susan Sienko Thomas, Cathleen E. Buckon, Erik K Henricson, Alina Nicorici, Michael D. Sussman

Research output: Contribution to journalArticle

42 Scopus citations

Abstract

In Duchenne muscular dystrophy, data directly linking changes in clinical outcome measures to patient-perceived well-being are lacking. This study evaluated the relationship between clinical outcome measures used in clinical trials of ambulatory Duchenne muscular dystrophy (Vignos functional grade, quantitative knee extension strength, timed functional performance measures, and gait velocity) and 2 health-related quality of life measures-the Pediatric Outcomes Data Collection Instrument and Pediatric Quality of Life Inventory-in 52 ambulatory Duchenne muscular dystrophy subjects and 36 controls. Those with the disease showed significant decrements in parent proxy-reported health-related quality of life measures versus controls across all domains. The Pediatric Outcomes Data Collection Instrument transfers/basic mobility and sports/ physical function and the Pediatric Quality of Life Inventory physical functioning domains had significant associations with age (and hence disease progression) and traditional clinical outcome measures employed in clinical trials of ambulatory boys with Duchenne muscular dystrophy. Selected domains of the Pediatric Outcomes Data Collection Instrument and generic Pediatric Quality of Life Inventory are potential patient-reported outcome measures for clinical trials in ambulatory individuals with the disease.

Original languageEnglish (US)
Pages (from-to)1130-1144
Number of pages15
JournalJournal of Child Neurology
Volume25
Issue number9
DOIs
StatePublished - Sep 2010

Keywords

  • clinical outcome measures
  • clinical trials
  • Duchenne muscular dystrophy
  • health-related quality of life
  • Pediatric Outcomes Data Collection Instrument (PODCI)
  • Pediatric Quality of Life Inventory (PedsQL)

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health

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