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Published on: March 27, 2014
Natural History of Steroid-Treated Young Boys With Duchenne Muscular Dystrophy Using the NSAA, 100m, and Timed
Natalie F Miller1, Lindsay N Alfano1, Megan A Iammarino1
1Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, Ohio.
Insights
This study defines the natural motor development in young boys with Duchenne muscular dystrophy (DMD) using standardized tests. These findings provide a baseline for evaluating future DMD treatments in early-intervention clinical trials.
Area of Science:
- Pediatric Neurology
- Clinical Research
- Biostatistics
Background:
- Duchenne muscular dystrophy (DMD) impacts motor development from early childhood.
- Early intervention is crucial for maximizing treatment efficacy in pediatric DMD.
- Understanding the natural history of motor maturation in young boys with DMD is essential for assessing treatment effects.
Purpose of the Study:
- To establish the natural history of motor function in boys aged 3 to 8 years with Duchenne muscular dystrophy.
- To provide normative data for key motor assessments in this cohort.
- To facilitate the interpretation of treatment effects in future clinical trials.
Main Methods:
- Evaluated 162 boys with DMD aged 3.1–7.9 years on glucocorticoids.
- Utilized North Star Ambulatory Assessment (NSAA), 100-meter timed test (100m), 10-meter walk/run (10m), time to rise (Rise), and 4-stair climb (4SC).
- Collected data during routine clinical visits; longitudinal data analyzed in a subcohort.
Main Results:
- Presented baseline functional data for boys with DMD aged 3.1 to 7.9 years.
- Detailed motor assessment results (NSAA, 100m, 10m, Rise, 4SC) across the evaluated age range.
- Longitudinal data provided insights into motor maturation patterns within the cohort.
Conclusions:
- The study establishes baseline motor function data for corticosteroid-treated boys with DMD.
- These findings are critical for comparing outcomes in ongoing and future DMD therapeutic interventions.
- The data supports the development of more effective early-stage DMD treatment strategies.
Introduction:
Clinical trials targeting younger cohorts of boys with Duchenne muscular dystrophy are necessary as earlier intervention may maximize treatment effect. Boys with Duchenne muscular dystrophy often have gross motor delays very early in life, and although they gain skills, they are on a lower trajectory than typical peers. Quantifying the natural rate of motor maturation in Duchenne muscular dystrophy from an early age permits identification of deviations from the expected trajectory related to treatment effects.
Methods:
The purpose of our study was to define the natural history in boys aged from ≥3 to <8 years using the North Star Ambulatory Assessment (NSAA), 100-meter timed test (100m), 10-meter walk/run (10m), time to rise (Rise), and 4-stair climb (4SC). Assessments were completed as standard of care during regularly scheduled clinic visits.
Results:
One hundred sixty-two boys with DMD aged 3.1 to 7.9 years on glucocorticoids were evaluated using one or more of the following tests as appropriate for age: NSAA (N = 158; 3.1-7.9 years), 100m (N = 131; 3.4-7.9 years), 10m (N = 162; 3.1-7.9 years), Rise (N = 160; 3.1-7.9 years), and 4SC (N = 153; 3.1-7.9 years). Longitudinal data are presented by age in a subcohort (N = 64).
Conclusions:
Our study documents the baseline function of boys with DMD who are being treated with corticosteroids. These data will be useful to compare ongoing and future therapeutic intervention(s) for DMD.
