Abstract
Introduction: Tests of ambulatory function are common clinical trial endpoints in Duchenne muscular dystrophy (DMD). Using these tests, the ImagingDMD study has generated a large data set that can describe the contemporary natural history of DMD in 5–12.9-year-olds. Methods: Ninety-two corticosteroid-treated boys with DMD and 45 controls participated in this longitudinal study. Participants performed the 6- minute walk test (6MWT) and timed function tests (TFT: 10-m walk/run, climbing 4 stairs, supine to stand). Results: Boys with DMD had impaired functional performance even at 5–6.9 years old. Boys older than 7 had significant declines in function over 1 year for 10-m walk/run and 6MWT. Eighty percent of participants could perform all functional tests at 9 years old. TFTs appear to be slightly more responsive and predictive of disease progression than the 6MWT in 7–12.9 year olds. Discussion: This study provides insight into the contemporary natural history of key functional endpoints in DMD.
Original language | English (US) |
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Pages (from-to) | 631-638 |
Number of pages | 8 |
Journal | Muscle and Nerve |
Volume | 58 |
Issue number | 5 |
DOIs | |
State | Published - Nov 2018 |
Keywords
- 6-minute walk test
- Ambulatory function
- Duchenne muscular dystrophy
- Functional endpoints
- Loss of ambulation
- Outcome measures
ASJC Scopus subject areas
- Physiology
- Clinical Neurology
- Cellular and Molecular Neuroscience
- Physiology (medical)