The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study

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

Research output: Contribution to journalArticle

140 Scopus citations

Abstract

Introduction: An international clinical trial enrolled 174 ambulatory males ≥5 years old with nonsense mutation Duchenne muscular dystrophy (nmDMD). Pretreatment data provide insight into reliability, concurrent validity, and minimal clinically important differences (MCIDs) of the 6-minute walk test (6MWT) and other endpoints. Methods: Screening and baseline evaluations included the 6-minute walk distance (6MWD), timed function tests (TFTs), quantitative strength by myometry, the PedsQL, heart rate-determined energy expenditure index, and other exploratory endpoints. Results: The 6MWT proved feasible and reliable in a multicenter context. Concurrent validity with other endpoints was excellent. The MCID for 6MWD was 28.5 and 31.7 meters based on 2 statistical distribution methods. Conclusions: The ratio of MCID to baseline mean is lower for 6MWD than for other endpoints. The 6MWD is an optimal primary endpoint for Duchenne muscular dystrophy (DMD) clinical trials that are focused therapeutically on preservation of ambulation and slowing of disease progression.

Original languageEnglish (US)
Pages (from-to)357-368
Number of pages12
JournalMuscle and Nerve
Volume48
Issue number3
DOIs
StatePublished - Sep 2013

Keywords

  • 6-minute walk test
  • Ambulation
  • Duchenne muscular dystrophy
  • Energy expenditure index
  • Muscular dystrophy
  • Myometry
  • Natural history
  • PedsQL
  • Timed function test

ASJC Scopus subject areas

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

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