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Updated: Jun 4, 2025
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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
10.3K
Relationship Between Quantitative Magnetic Resonance Imaging Measures and Functional Changes in Patients With
Sarah P Sherlock1, Allison McCrady2, Jeffrey Palmer1
1Pfizer Inc, Cambridge, Massachusetts, USA.
Muscle & Nerve
|December 23, 2024
Summary
Muscle MRI biomarkers like lean muscle volume and fat infiltration show strong correlations with functional outcomes in Duchenne muscular dystrophy (DMD). These findings support MRI
Area of Science:
- Biomedical imaging
- Neuromuscular disorders
- Clinical trial methodology
Background:
- Duchenne muscular dystrophy (DMD) requires improved monitoring methods for clinical trials.
- Muscle magnetic resonance imaging (MRI) offers potential biomarkers for disease progression.
Purpose of the Study:
- To evaluate changes in muscle MRI parameters (lean muscle volume, fat fraction, fat infiltration) in DMD patients.
- To assess the relationship between these MRI parameters and functional outcomes.
Main Methods:
- Post hoc analysis of MRI and functional data from a domagrozumab clinical study (NCT02310763).
- Dixon MRI technique used to assess adductors, hamstrings, and quadriceps at baseline, week 49, and week 97.
Main Results:
- Increased lean muscle volume (LMV) positively correlated with function, particularly in quadriceps and adductors.
- Increased muscle fat fraction (MFF) and fat infiltration (MFI) negatively correlated with function across all evaluated muscle groups.
- High LMV and low MFI were associated with preserved ambulatory function.
Conclusions:
- Muscle MRI parameters (LMV, MFF, MFI) are reliable indicators of DMD progression.
- These MRI biomarkers show potential as surrogate endpoints in clinical trials for DMD.
