Abstract Details

Title Associations Between Deflazacort Versus Prednisone/Prednisolone and Markers of Disease Progression in Clinically Important Subgroups of Patients with Duchenne Muscular Dystrophy

Topic Child Neurology and Developmental Neurology

Presentation(s) P1 - Poster Session 1 (9:00 AM-5:00 PM)

Poster/Presentation
Number 135

Objective To compare clinical outcomes in Duchenne muscular dystrophy (DMD) by steroid type within subgroups defined by baseline age, ambulatory function, and steroid duration.

Background The standard of care for DMD includes steroids. At the same time, clinical outcomes remain heterogeneous among boys receiving steroid treatment. It is important to understand the extent to which steroid effects might vary across clinically distinct subgroups.

Design/Methods Placebo arms from four DMD clinical trials with assessments of 48-week change in six-minute walk distance (6MWD) as the primary outcome were studied (NCT01826487, NCT01865084, NCT00592553, NCT01254019). Mean changes in 6MWD and secondary ambulatory outcomes were compared between patients receiving daily deflazacort vs. daily prednisone, adjusting for baseline age, steroid duration, 6MWD, and rise time.

Results A total of n=328 patients were available across placebo arms, with n=231 receiving daily steroids (n=127 deflazacort; n=104 prednisone). Stable steroid use for ≥6 months pre-baseline was required in all but one study; all had mean prior steroid use > 1.5 years. Baseline characteristics were balanced across steroid groups. In the overall study population, deflazacort was associated with preservation of 35.4 meters of 6MWD over 48 weeks compared to prednisone (P<0.01). When assessing by subgroups, differences between deflazacort vs. prednisone were most pronounced among boys with the following baseline characteristics: aged ≥8 years (+44.5m, P<0.01), rise time ≥5 seconds (+41.3m, P<0.01) and steroid duration >3 years (+57.4m, P<0.01). Overall differences between steroid groups in timed function tests and in the linearized North Start Ambulatory Assessment were numerically consistent.

Conclusions Benefits of daily deflazacort compared to daily prednisone for preserving ambulatory function in DMD were most evident among patients who were older, had been on steroids longer, or were at a more progressed disease stage. These results add to the evidence for a potential cumulative benefit of deflazacort versus prednisone.

Authors/Disclosures
Craig McDonald, MD (UC Davis Dept. of PM&R) PRESENTER	Dr. McDonald has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Sarepta Therapeutics. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for PTC Therapeutics. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Solid Biosciences. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. McDonald 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 Therapeutics. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid Biosciences. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Edgewise Therapeutics. The institution of Dr. McDonald has received research support from Sarepta Therapeutics. The institution of Dr. McDonald has received research support from PTC Therapeutics. The institution of Dr. McDonald has received research support from Edgewise Therapeutics. The institution of Dr. McDonald has received research support from Capricor Therapeutics. The institution of Dr. McDonald has received research support from Italfarmaco. Dr. McDonald has received research support from NS Pharma. The institution of Dr. McDonald has received research support from NIH (NINDS). The institution of Dr. McDonald has received research support from Parent Project Muscular Dystrophy. The institution of Dr. McDonald has received research support from Muscular Dystrophy Association. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Member National Advisory Board for Medical Rehabilitation Research with NIH.
	No disclosure on file
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Claudio Santos	No disclosure on file
Richard A. Able, Jr., PhD	Dr. Able has received personal compensation for serving as an employee of Praxis Medicines. Dr. Able has received personal compensation for serving as an employee of PTC Therapeutics. Dr. Able has stock in Praxis Medicines. Dr. Able has received personal compensation in the range of $0-$499 for serving as a Employee with PTC therapeutics.
	No disclosure on file

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