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MR biomarkers predict clinical function in Duchenne muscular dystrophy

Alison M. Barnard, DPT, PhD

Alison M. Barnard, DPT, PhD

Alison Barnard has been a part of the ImagingNMD team since 2014. With training as a physical therapist from the University of Alabama at Birmingham, she joined the team as a University of Florida PhD student and gained specialized expertise in muscular dystrophy and magnetic resonance imaging (MRI) and spectroscopy (MRS) in skeletal muscle. Dr. Barnard is now a research assistant professor at the University of Florida and a study investigator with ImagingNMD. Her research, particularly in the application of MRI to monitor DMD, has been instrumental in the development of clinical trial methodologies and understanding of disease progression. Dr. Barnard has published several papers with the ImagingNMD team, including a manuscript that translates MRI knowledge to practical information for clinical physical therapists treating individuals with DMD. Within the ImagingNMD group, Dr. Barnard collects MRI and functional outcome data from participants with DMD and BMD, oversees biosample collection and storage, and manages genetic data information. She also has a special interest in respiratory muscle involvement in DMD, and Dr. Barnard helps lead study efforts in this area.

Alison M. Barnard, DPT, PhD,* Rebecca J. Willcocks, PhD,* William T. Triplett, BS, Sean C. Forbes, PhD,
Michael J. Daniels, ScD, Saptarshi Chakraborty, PhD, Donovan J. Lott, PT, PhD, Claudia R. Senesac, PT, PhD, Erika L. Finanger, MD, PhD, Ann T. Harrington, DPT, PhD, Gihan Tennekoon, MBBS, Harneet Arora, PT, PhD, Dah-Jyuu Wang, PhD, H. Lee Sweeney, PhD, William D. Rooney, PhD, Glenn A. Walter, PhD, and Krista Vandenborne, PT, PhD

Abstract

Objective

To investigate the potential of lower extremity magnetic resonance (MR) biomarkers to serve
as endpoints in clinical trials of therapeutics for Duchenne muscular dystrophy (DMD) by
characterizing the longitudinal progression of MR biomarkers over 48 months and assessing
their relationship to changes in ambulatory clinical function.

Methods

One hundred sixty participants with DMD were enrolled in this longitudinal, natural history
study and underwent MR data acquisition of the lower extremity muscles to determine muscle
fat fraction (FF) and MRI T2 biomarkers of disease progression. In addition, 4 tests of ambulatory function were performed. Participants returned for follow-up data collection at 12, 24,
36, and 48 months.

Results

Longitudinal analysis of the MR biomarkers revealed that vastus lateralis FF, vastus lateralis
MRI T2, and biceps femoris long head MRI T2 biomarkers were the fastest progressing
biomarkers over time in this primarily ambulatory cohort. Biomarker values tended to demonstrate a nonlinear, sigmoidal trajectory over time. The lower extremity biomarkers predicted
functional performance 12 and 24 months later, and the magnitude of change in an MR
biomarker over time was related to the magnitude of change in function. Vastus lateralis FF,
soleus FF, vastus lateralis MRI T2, and biceps femoris long head MRI T2 were the strongest
predictors of future loss of function, including loss of ambulation.

Conclusions

This study supports the strong relationship between lower extremity MR biomarkers and
measures of clinical function, as well as the ability of MR biomarkers, particularly those from
proximal muscles, to predict future ambulatory function and important clinical milestones.

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Null.
I can’t say enough positive things about the entire team. Everyone has consistently gone out of their way to accommodate us & support us. We’ve been participating for so many years that our annual trips feel like we’re reuniting with our good friends.
WY
A person wearing black tight and colorful shoes with flower on it, playing the game hopscotch outside, with their left food on the number three and their right foot up in the air.
Null.
Very important if we can eliminate intrusive biopsies as a necessary requirement for clinical trial evaluation. MRIs are a much preferred “gold standard”.
KT
A person wearing black tight and colorful shoes with flower on it, playing the game hopscotch outside, with their left food on the number three and their right foot up in the air.
Null.
The study has been very exciting. Discovering new tools that can help in the development of more studies and clinical trials for Becker muscular dystrophy. I feel like people are beginning to recognize BMD now.
JV

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