THE 6‐minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study. Issue 3 (26th June 2013)

• Record Type: Journal Article
• Authors: 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
• Is Part Of: Muscle & nerve. Volume 48:Issue 3(2013:Sep.)
• Page Start: 343
• Page End: 356
• Publication Date: 2013-06-26
• Subjects: Neuromuscular diseases -- Periodicals; Muscles -- Periodicals; Nerves -- Periodicals; 616.74
• DOI: 10.1002/mus.23902
• Languages: English
• ISSNs: 0148-639X

Abstract

<abstract abstract-type="main"> <title>ABSTRACT</title> <p> <italic>Introduction</italic>: 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 (<italic>N</italic> = 57) provided insight into the natural history of the 6‐minute walk test (6MWT) and other endpoints. <italic>Methods</italic>: 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 &lt;350 meters was associated with greater functional decline, and loss of ambulation was only seen in those with baseline 6MWD &lt;325 meters. Only 1 of 42 (2.3%) subjects able to stand from supine lost ambulation. <italic>Conclusion</italic>: Findings confirm the clinical meaningfulness of the 6MWD as the most accepted primary clinical endpoint in ambulatory DMD trials. <italic>Muscle Nerve</italic><bold>48</bold>: 343–356, 2013</p> </abstract>