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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
Author(s) -
McDonald Craig M.,
Henricson Erik K.,
Abresch R. Ted,
Florence Julaine,
Eagle Michelle,
Gappmaier Eduard,
Glanzman Allan M.,
Spiegel Robert,
Barth Jay,
Elfring Gary,
Reha Allen,
Peltz Stuart W.
Publication year - 2013
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.23905
Subject(s) - minimal clinically important difference , medicine , duchenne muscular dystrophy , context (archaeology) , physical therapy , physical medicine and rehabilitation , clinical endpoint , clinical trial , concurrent validity , reliability (semiconductor) , muscular dystrophy , randomized controlled trial , surgery , patient satisfaction , paleontology , power (physics) , physics , quantum mechanics , internal consistency , biology
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. Muscle Nerve 48 : 357–368, 2013