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Updated: Feb 18, 2026
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Timed function tests, motor function measure, and quantitative thigh muscle MRI in ambulant children with Duchenne
Simone Schmidt1, Patricia Hafner2, Andrea Klein3
1Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland; Department of Neurology, University of Basel Hospital, Basel, Switzerland.
Insights
This study found that clinical assessments and muscle MRI are comparable in measuring Duchenne muscular dystrophy severity. Both methods correlate with disease progression, aiding in the development of new therapeutic agents.
Area of Science:
- Biomedical Engineering
- Neurology
- Radiology
Background:
- Duchenne muscular dystrophy (DMD) treatment development requires sensitive outcome measures.
- Clinical assessments and quantitative muscle MRI are potential tools for evaluating treatment efficacy.
Purpose of the Study:
- To investigate the relationship between clinical assessments and quantitative muscle MRI in ambulant DMD patients.
- To determine the sensitivity of these measures to disease progression and age.
Main Methods:
- Cross-sectional analysis of ambulant DMD patients (aged 6.5-10.8 years).
- Clinical assessments included the 6-minute walk test and Motor Function Measure.
- Quantitative muscle MRI measured fat fraction and T2 relaxation time in thigh muscles.
Main Results:
- Clinical assessments were highly inter-correlated and showed no age correlation.
- Quantitative muscle MRI values correlated significantly with all clinical assessments, especially in extensor muscles.
- Muscle MRI values showed a strong correlation with patient age, unlike clinical assessments.
Conclusions:
- Motor Function Measure and timed function tests similarly reflect DMD disease severity.
- Quantitative muscle MRI effectively quantifies fatty muscle degeneration and correlates with clinical measures.
- Combined clinical and MRI assessments offer comprehensive insights into DMD progression for therapeutic development.
Abstract:
The development of new therapeutic agents for the treatment of Duchenne muscular dystrophy has put a focus on defining outcome measures most sensitive to capture treatment effects. This cross-sectional analysis investigates the relation between validated clinical assessments such as the 6-minute walk test, motor function measure and quantitative muscle MRI of thigh muscles in ambulant Duchenne muscular dystrophy patients, aged 6.5 to 10.8 years (mean 8.2, SD 1.1). Quantitative muscle MRI included the mean fat fraction using a 2-point Dixon technique, and transverse relaxation time (T2) measurements. All clinical assessments were highly significantly inter-correlated with p < 0.001. The strongest correlation with the motor function measure and its D1-subscore was shown by the 6-minute walk test. Clinical assessments showed no correlation with age. Importantly, quantitative muscle MRI values significantly correlated with all clinical assessments with the extensors showing the strongest correlation. In contrast to the clinical assessments, quantitative muscle MRI values were highly significantly correlated with age. In conclusion, the motor function measure and timed function tests measure disease severity in a highly comparable fashion and all tests correlated with quantitative muscle MRI values quantifying fatty muscle degeneration.
