Abstract Details

Title Deflazacort Or Prednisone Treatment for Duchenne Muscular Dystrophy (DMD) Real-World Outcomes at Cincinnati Children’s Hospital Medical Center (CCHMC)

Topic Child Neurology and Developmental Neurology

Presentation(s) P15 - Poster Session 15 (12:00 PM-1:00 PM)

Poster/Presentation
Number 5-012

Objective This study assessed real-world data for deflazacort or prednisone steroid treatment, and ambulatory, pulmonary, cardiac, growth and bone-health outcomes.

Background Corticosteroids are currently the standard of care for the treatment of patients with Duchenne muscular dystrophy.

Design/Methods Over 200 boys at CCHMC were included in the analysis. About 75% received deflazacort (~95% daily) and 13% prednisone (~75% daily). The remaining 12% switched from prednisone to deflazacort (average duration 3.1 years on prednisone and 2.5 years on deflazacort).

Results On average, after accounting for age and steroid duration in a regression model, patients on deflazacort compared with prednisone had: 0.59 stairs/second greater 4-stair climb velocity, 4.6 points higher North Star Ambulatory Assessment total score and 9.9% higher forced vital capacity %-predicted (P<0.05 for each). Total body mass was lower by 6.9kg and height was lower by 6.2cm; % lean body mass was higher by 4.4%. No significant difference was observed in whole-body bone mineral density or in left ventricular ejection fraction. In Kaplan-Meier analyses, by age 15 (age 20), 56.4% (85.1%) of prednisone-initiated patients used a wheelchair as their primary means of mobility, compared to 43.7% (78.7%) of deflazacort-initiated patients (P<0.01). By age 15 (20), 13.7% (64.8%) of prednisone-initiated patients had scoliosis compared to 8.9% (33.7%) of deflazacort-initiated patients (P=0.05). These differences persisted after adjusting for age at steroid initiation. In sensitivity analyses, risk of scoliosis and wheelchair use among switchers was numerically lower vs consistent prednisone but higher versus consistent deflazacort.

Conclusions

This study adds evidence associating deflazacort use with greater functional preservation relative to prednisone, and further indicates concurrent preservation of lean body mass and delay of scoliosis.

Authors/Disclosures
PRESENTER	No disclosure on file
Claudio Santos	No disclosure on file
Panayiota Trifillis	Panayiota Trifillis has received personal compensation for serving as an employee of PTC Therapeutics. Panayiota Trifillis has received stock or an ownership interest from PTC Therapeutics, Inc.. Panayiota Trifillis has received intellectual property interests from a discovery or technology relating to health care.
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
Cuixia Tian, MD (Cincinnati Childrens Hospital Medical Center)	Dr. Tian has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Tian has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Tian has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Itf. Dr. Tian has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regenxbio. Dr. Tian has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid Bioscience. The institution of Dr. Tian has received research support from MDA. The institution of Dr. Tian has received research support from NIH.
Brenda L. Wong, MD (University of Massachusetts Medical School)	No disclosure on file

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Abstract Details