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; PTC124‐GD‐007‐DMD Study Group

doi:10.1002/mus.23902

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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

Journal article

Open access

Peer reviewed

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, …

Muscle & nerve, Vol.48(3), pp.343-356

09/2013

DOI: 10.1002/mus.23902

PMCID: PMC3824082

PMID: 23681930

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Abstract

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. Evaluations performed every 6 weeks included the 6-minute walk distance (6MWD), timed function tests (TFTs), and quantitative strength using hand-held myometry. 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. Findings confirm the clinical meaningfulness of the 6MWD as the most accepted primary clinical endpoint in ambulatory DMD trials.

Electromyography

Predictive Value of Tests

Glucocorticoids - therapeutic use

Double-Blind Method

Muscular Dystrophy, Duchenne - drug therapy

Humans

Child, Preschool

Male

Oxadiazoles - therapeutic use

Walking - physiology

Exercise Test

Time Factors

Muscular Dystrophy, Duchenne - physiopathology

Adolescent

Observation

Outcome Assessment, Health Care

Child

Hand Strength - physiology

Longitudinal Studies

Details

Title: Subtitle: The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study
Creators: Craig M McDonald - Department of Physical Medicine and Rehabilitation, University of California Davis School of Medicine, Davis, California, 95817, USA
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
PTC124‐GD‐007‐DMD Study Group
Contributors: Katherine D Mathews (Contributor) - University of Iowa, Stead Family Department of Pediatrics
Resource Type: Journal article
Publication Details: Muscle & nerve, Vol.48(3), pp.343-356
DOI: 10.1002/mus.23902
PMID: 23681930
PMCID: PMC3824082
NLM abbreviation: Muscle Nerve
ISSN: 0148-639X
eISSN: 1097-4598
Publisher: United States
Grant note: MR/K000608/1 / Medical Research Council
Language: English
Date published: 09/2013
Academic Unit: Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
Record Identifier: 9984020656902771

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