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Muscle Nerve. 2013 Sep;48(3):343-56. doi: 10.1002/mus.23902. Epub 2013 Jun 26.

The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.

Author information

1
Department of Physical Medicine and Rehabilitation, University of California Davis School of Medicine, Davis, California, 95817, USA.

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.

KEYWORDS:

6-minute walk test; Duchenne muscular dystrophy; ambulation; dystrophinopathy; myometry; natural history; prediction of loss of function; timed function tests

PMID:
23681930
PMCID:
PMC3824082
DOI:
10.1002/mus.23902
[Indexed for MEDLINE]
Free PMC Article

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