THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study

Craig M McDonald, Erik K Henricson, R. Ted Abresch, Julaine M. Florence, Michelle Eagle, Eduard Gappmaier, Allan M. Glanzman, Robert Spiegel, Jay Barth, Gary Elfring, Allen Reha, Stuart Peltz

Research output: Contribution to journalArticle

156 Scopus citations

Abstract

Introduction: Duchenne muscular dystrophy (DMD) subjects ≥5 years with nonsense mutations were followed for 48 weeks in a multicenter, randomized, double-blind, placebo-controlled trial of ataluren. Placebo arm data (N = 57) provided insight into the natural history of the 6-minute walk test (6MWT) and other endpoints. Methods: Evaluations performed every 6 weeks included the 6-minute walk distance (6MWD), timed function tests (TFTs), and quantitative strength using hand-held myometry. Results: Baseline age (≥7 years), 6MWD, and selected TFT performance are strong predictors of decline in ambulation (Δ6MWD) and time to 10% worsening in 6MWD. A baseline 6MWD of <350 meters was associated with greater functional decline, and loss of ambulation was only seen in those with baseline 6MWD <325 meters. Only 1 of 42 (2.3%) subjects able to stand from supine lost ambulation. Conclusion: Findings confirm the clinical meaningfulness of the 6MWD as the most accepted primary clinical endpoint in ambulatory DMD trials.

Original languageEnglish (US)
Pages (from-to)343-356
Number of pages14
JournalMuscle and Nerve
Volume48
Issue number3
DOIs
StatePublished - Sep 2013

Keywords

  • 6-minute walk test
  • Ambulation
  • Duchenne muscular dystrophy
  • Dystrophinopathy
  • Myometry
  • Natural history
  • Prediction of loss of function
  • Timed function tests

ASJC Scopus subject areas

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

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    McDonald, C. M., Henricson, E. K., Abresch, R. T., Florence, J. M., Eagle, M., Gappmaier, E., Glanzman, A. M., Spiegel, R., Barth, J., Elfring, G., Reha, A., & Peltz, S. (2013). THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study. Muscle and Nerve, 48(3), 343-356. https://doi.org/10.1002/mus.23902