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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.
- Source :
-
Muscle & nerve [Muscle Nerve] 2013 Sep; Vol. 48 (3), pp. 343-56. Date of Electronic Publication: 2013 Jun 26. - Publication Year :
- 2013
-
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.<br />Methods: Evaluations performed every 6 weeks included the 6-minute walk distance (6MWD), timed function tests (TFTs), and quantitative strength using hand-held myometry.<br />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.<br />Conclusion: Findings confirm the clinical meaningfulness of the 6MWD as the most accepted primary clinical endpoint in ambulatory DMD trials.<br /> (Copyright © 2013 Wiley Periodicals, Inc.)
- Subjects :
- Adolescent
Child
Child, Preschool
Double-Blind Method
Electromyography
Glucocorticoids therapeutic use
Hand Strength physiology
Humans
Longitudinal Studies
Male
Muscular Dystrophy, Duchenne drug therapy
Observation
Oxadiazoles therapeutic use
Predictive Value of Tests
Time Factors
Exercise Test
Muscular Dystrophy, Duchenne physiopathology
Outcome Assessment, Health Care
Walking physiology
Subjects
Details
- Language :
- English
- ISSN :
- 1097-4598
- Volume :
- 48
- Issue :
- 3
- Database :
- MEDLINE
- Journal :
- Muscle & nerve
- Publication Type :
- Academic Journal
- Accession number :
- 23681930
- Full Text :
- https://doi.org/10.1002/mus.23902