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

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.

• Authors: Craig M McDonald, Erik K Henricson, R Ted Abresch, Julaine M Florence, Michelle Eagle, Eduard Gappmaier, Allan M Glanzman, PTC124-GD-007-DMD Study Group, Robert Spiegel, Jay Barth, Gary Elfring, Allen Reha, Stuart Peltz
• Journal: Muscle & nerve (Muscle Nerve) Vol. 48 Issue 3 Pg. 343-56 (Sep 2013) ISSN: 1097-4598 [Electronic] United States
• PMID: 23681930 (Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial)
• Copyright: Copyright © 2013 Wiley Periodicals, Inc.

Chemical References

• Glucocorticoids
• Oxadiazoles
• ataluren

Topics

• Adolescent
• Child
• Child, Preschool
• Double-Blind Method
• Electromyography
• Exercise Test
• Glucocorticoids (therapeutic use)
• Hand Strength (physiology)
• Humans
• Longitudinal Studies
• Male
• Muscular Dystrophy, Duchenne (drug therapy, physiopathology)
• Observation
• Outcome Assessment, Health Care
• Oxadiazoles (therapeutic use)
• Predictive Value of Tests
• Time Factors
• Walking (physiology)