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THE 6‐minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study
Author(s) -
Mcdonald Craig M.,
Henricson Erik K.,
Abresch R. Ted,
Florence Julaine M.,
Eagle Michelle,
Gappmaier Eduard,
Glanzman Allan M.,
Spiegel Robert,
Barth Jay,
Elfring Gary,
Reha Allen,
Peltz Stuart
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.23902
Subject(s) - duchenne muscular dystrophy , medicine , supine position , clinical endpoint , ambulatory , placebo , physical therapy , natural history , clinical trial , multicenter trial , physical medicine and rehabilitation , muscular dystrophy , randomized controlled trial , multicenter study , alternative medicine , pathology
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. Muscle Nerve 48 : 343–356, 2013