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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
10.9K
Longitudinal community walking activity in Duchenne muscular dystrophy
Eileen G Fowler1, Loretta A Staudt1, Kent R Heberer1
1Department of Orthopaedic Surgery, University of California Los Angeles, 22-70 Rehab Center, 1000 Veteran Avenue, Los Angeles, California, 90095-1795, USA.
Muscle & Nerve
|July 11, 2017
Summary
Step activity monitors effectively measured community ambulation in Duchenne muscular dystrophy (DMD) boys. These real-world data are sensitive to changes over time and can inform clinical trials.
Area of Science:
- Biomedical Engineering
- Neuromuscular Disorders
- Clinical Trials
Background:
- Duchenne muscular dystrophy (DMD) natural history studies have lacked community ambulation metrics.
- Assessing real-world mobility is crucial for understanding disease progression and treatment efficacy in DMD.
Purpose of the Study:
- To quantify community ambulation in boys with DMD using step activity (SA) monitors.
- To determine the validity and sensitivity of SA monitors by comparing them with 10-meter walk/run (10mWR) speed.
Main Methods:
- 42 boys (ages 4-16) with DMD were enrolled serially for up to 5 years.
- Step activity monitors were used to quantify community ambulation.
- Data were analyzed using repeated-measures mixed models and compared with 10mWR speed.
Main Results:
- Significant age-related declines were observed in average strides per day and percentage of strides at various intensity rates (P < 0.05).
- 10mWR speed showed significant correlations with high and low stride rates at baseline (P < 0.05).
- SA outcomes demonstrated sensitivity to annual changes, with variations based on age group.
Conclusions:
- Community ambulation data derived from SA monitors offer valid and sensitive real-world measures for DMD.
- These metrics can provide valuable insights for informing the design and outcomes of clinical trials in DMD.
