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 journalArticlepeer-review

202 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
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

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