Abstract Details

Title Developing Tools to Assess Changes Associated with Aerobic Exercise in Boys with Duchenne Muscular Dystrophy

Topic Child Neurology and Developmental Neurology

Presentation(s) P1 - Poster Session 1 (5:30 PM-6:30 PM)

Poster/Presentation
Number 7-069

Objective To develop clinical, laboratory, and survey data points to understand how aerobic exercise impacts boys with Duchenne Muscular Dystrophy.

Background Aerobic exercise has been proposed as a supportive measure to improve health in boys with DMD. Tools to assess acute and long-term changes associated with aerobic exercise in boys with DMD do not exist. Standardized assessments are essential for conducting future exercise trials.

Design/Methods

Patients were recruited during clinic, underwent standard of care (body mass index (BMI) and Brooke Functional Assessment) in addition to providing a blood sample (serum glucose) and completing surveys (Quality of Life, Ambulatory Survey, and Exercise Barriers). This study was approved by our institutional IRB.

Results 44 patients with DMD were recruited (mean age of 9 years, 59% ambulatory, 25% nonambulatory, 16% transitioning). There was a direct correlation (R² = 0.74) between quality of life (Pediatric Quality of Life Survey) and ability to ambulate (Ambulatory Survey). Exercise barriers were minimal in both early ambulatory (Brooke score 1-2) and non-ambulatory boys (Brooke score 9). Exercise barriers were increased in patients transitioning (Brooke score 3-8) from ambulatory to non-ambulatory status in 3 out of the 8 categories: I don’t think it will help, I fear it will injure my child, and my child does not have the energy. BMI levels were significantly decreased (p < 0.05) in ambulatory DMD boys (18% BMI) as compared to non-ambulatory (27% BMI) and weakly correlated to Brooke score (R² = 0.31). Serum glucose levels showed no correlation with ambulatory survey scores (R² = 0.03), and Brooke (R² = 0.00), but increased with increasing age (R² =0.31).

Conclusions Delaying onset of loss of ambulation in boys with DMD will improve their quality of life and BMI. Boys who are transitioning between ambulatory and nonambulatory status need increased support to continue to exercise.

Authors/Disclosures
Aaron Zelikovich, MD PRESENTER	Dr. Zelikovich has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genentech.
	No disclosure on file
	No disclosure on file
	No disclosure on file
Vamshi Rao, MD (Ann & Robert H Lurie Children's Hospital)	No disclosure on file
Nancy L. Kuntz, MD, FAAN (Ann & Robert H Lurie Children'S Hospital of Chicago)	Dr. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Kuntz has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenyx. Dr. Kuntz has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Audentes Therapeutics. Dr. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Kuntz has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Kuntz has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. Dr. Kuntz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. The institution of Dr. Kuntz has received research support from Argenx. The institution of Dr. Kuntz has received research support from Astellas. The institution of Dr. Kuntz has received research support from Biogen. The institution of Dr. Kuntz has received research support from Catalyst. The institution of Dr. Kuntz has received research support from Genentech. The institution of Dr. Kuntz has received research support from Scholar Rock. The institution of Dr. Kuntz has received research support from Regenx-Bio. The institution of Dr. Kuntz has received research support from Solid Biosciences. The institution of Dr. Kuntz has received research support from AMO. The institution of Dr. Kuntz has received research support from Novartis. The institution of Dr. Kuntz has received research support from BioHaven. Dr. Kuntz has received personal compensation in the range of $50,000-$99,999 for serving as a Sarepta Express post gene therapy advisor with Sarepta.

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