Associations Between Deflazacort Versus Prednisone/Prednisolone and Markers of Disease Progression in Clinically Important Subgroups of Patients with Duchenne Muscular Dystrophy (P1-1.Virtual)
Abstract
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.
Disclosure: Dr. McDonald has received personal compensation in the range of $10,000-$49,999 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 $500-$4,999 for serving as a Consultant for Astellas. Dr. McDonald has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Capricor Therapeutics. Dr. McDonald has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Catabasis. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epirium Bio. Dr. McDonald has received personal compensation in the range of $0-$499 for serving as a Consultant for Italfarmaco. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Santhera Pharmaceuticals. 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 $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC Therapeutics. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Capricor. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis Pharmaceuticals. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Santhera Pharmaceuticals. 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 Santhera Pharmaceuticals. The institution of Dr. McDonald has received research support from Astellas. The institution of Dr. McDonald has received research support from Capricor Therapeutics. The institution of Dr. McDonald has received research support from Catabasis. The institution of Dr. McDonald has received research support from Epirium Bio. 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 Pfizer. The institution of Dr. McDonald has received research support from NIH (NINDS). The institution of Dr. McDonald has received research support from NIDILRR. The institution of Dr. McDonald has received research support from Department of Defense. 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. The institution of Jessica Marden has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Analysis Group, Inc. The institution of Jessica Marden has received research support from PTC Therapeutics. The institution of Henry Lane has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Analysis Group. Ms. Zhang has received personal compensation for serving as an employee of Analysis Group. Ms. Nguyen has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Analysis Group, Inc. Claudio Santos has received personal compensation for serving as an employee of PTC. Claudio Santos has received stock or an ownership interest from PTC. Dr. Able has received stock or an ownership interest from PTC Therapeutis. Dr. Able has received personal compensation in the range of $0-$499 for serving as a Employee with PTC therapeutics. The institution of James Signorovitch has received research support from PTC Therapeutics.
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Neurology®
Volume 98 • Number 18_supplement • May 3, 2022
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Published online: May 3, 2022
Published in print: May 3, 2022
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