Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy

Jill E. Mayhew, Julaine M. Florence, Thomas P. Mayhew, Erik K Henricson, Robert T. Leshner, Robert J. McCarter, Diana M. Escolar

Research output: Contribution to journalArticle

86 Scopus citations

Abstract

We studied the reliability of a series of endpoints in an evaluation of subjects with Duchenne muscular dystrophy (DMD). The endpoints included quantitative muscle tests (QMTs), timed function tests, forced vital capacity (FVC), and manual muscle tests (MMT). Thirty-one ambulatory subjects with DMD (mean age 8.9 years; range 5-16 years) were evaluated at eight sites by 15 newly trained evaluators as a test of interrater reliability of outcome measures. Both total QMT score [intraclass correlation coefficient (ICC) 0.96] and individual QMT assessments (ICC 0.85-0.96) were highly reliable. Forced vital capacity and all timed function tests were also highly reliable (ICC 0.97-0.99). MMT was the least reliable assessment method (ICC 0.61). These data suggest that primary surrogate outcome measures in large multicenter clinical trials in DMD should use QMT, FVC, or time function tests to obtain maximum power and greatest sensitivity.

Original languageEnglish (US)
Pages (from-to)36-42
Number of pages7
JournalMuscle and Nerve
Volume35
Issue number1
DOIs
StatePublished - Jan 2007
Externally publishedYes

Keywords

  • Duchenne muscular dystrophy
  • Interrater reliability
  • Pediatric neuromuscular testing
  • Quantitative muscle testing (QMT)

ASJC Scopus subject areas

  • Physiology
  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)

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  • Cite this

    Mayhew, J. E., Florence, J. M., Mayhew, T. P., Henricson, E. K., Leshner, R. T., McCarter, R. J., & Escolar, D. M. (2007). Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy. Muscle and Nerve, 35(1), 36-42. https://doi.org/10.1002/mus.20654