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Updated: May 7, 2026
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Patterns of growth in ambulatory males with Duchenne muscular dystrophy
Nancy A West1, Michele L Yang, David A Weitzenkamp
1Department of Epidemiology, Colorado School of Public Health, University of Colorado, Aurora, CO.
Insights
Growth charts for boys with Duchenne muscular dystrophy (DMD) show they are shorter and have more extreme weights and BMIs than healthy boys. These standards aid in monitoring growth and treatment responses.
Area of Science:
- Pediatric Endocrinology
- Neuromuscular Disorders
- Growth and Development
Background:
- Duchenne muscular dystrophy (DMD) affects muscle growth and development.
- Standard growth charts may not accurately reflect growth patterns in children with DMD.
- Ambulatory, steroid-naïve males with DMD require specific growth references.
Purpose of the Study:
- Establish weight-for-age, height-for-age, and BMI-for-age growth reference standards for ambulatory, steroid-naïve males aged 2-12 years with DMD.
- Compare DMD growth curves to the 2000 Centers for Disease Control and Prevention (CDC) growth charts.
- Provide tools for monitoring growth and treatment in pediatric DMD patients.
Main Methods:
- Retrospective analysis of 1877 weight and 1544 height measurements from 513 males with DMD (1985-2010).
- Case classification based on ambulation and symptom onset criteria for DMD.
- Growth charts constructed using 5th, 10th, 25th, 50th, 75th, and 90th percentiles with smoothing procedures.
Main Results:
- Developed growth curves for ambulatory, steroid-naïve males with DMD.
- DMD males exhibit shorter stature compared to the general US pediatric male population.
- DMD males show a tendency towards extreme values in weight and body mass index.
Conclusions:
- Growth charts for DMD provide essential standards for monitoring growth patterns.
- Facilitates early detection of abnormal growth in pediatric DMD.
- Assists in evaluating the efficacy of corticosteroid treatment in DMD.
Objective:
To provide weight-for-age, height-for-age, and body mass index-for-age growth reference standards for ambulatory, steroid-naïve males, ages 2-12 years, with Duchenne muscular dystrophy (DMD) and to compare these growth curves to the 2000 Centers for Disease Control and Prevention growth charts for boys, which serve as references of physical size and growth for the general male pediatric population in the US.
Study Design:
Through a multi-state population-based surveillance of individuals with muscular dystrophy, a total of 1877 weight and 1544 height measurements ascertained during 1985-2010 from 513 males with DMD were obtained retrospectively from medical record review. Cases were classified as DMD if loss of ambulation occurred before the 12th birthday or, if younger than 12 years and still ambulating, the earliest symptoms of dystrophinopathy occurred before the 6th birthday. Each growth chart was constructed using 5 percentiles: 10th, 25th, 50th, 75th, and 90th. Smoothing procedures were applied in 2 stages to the irregular plots of the empirical percentile values.
Results:
A set of growth curves, derived from a large cohort of male youth with DMD, are presented. These curves demonstrate that DMD males are shorter and tend to the extremes of weight and body mass index compared with the general male pediatric population in the US.
Conclusion:
Charts representing the pattern of growth in ambulatory, steroid-naïve males with DMD can facilitate monitoring of growth and early detection of unusual growth patterns. Use of these growth standards also will assist in monitoring responses to corticosteroid treatment.
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