The 6-minute Walk Test and Other Endpoints in Duchenne Muscular Dystrophy: Longitudinal Natural History Observations over 48 Weeks from a Multicenter Study

Overview

Journal Muscle Nerve

Date 2013 May 18

PMID 23681930

Citations 140

Authors

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

Affiliations

Soon will be listed here.

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.

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