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Abstract Details

Real-World Reasons for Steroid Switching and Associated Clinical Outcomes Among Patients with Dystrophinopathies in the US: a Nationwide Review of Medical Records
Child Neurology and Developmental Neurology
Child Neurology and Developmental Neurology Posters (7:00 AM-5:00 PM)
070
To describe reasons for switching from prednisone/prednisolone to deflazacort, and clinical outcomes, among US patients with dystrophinopathies. 
Corticosteroid treatment with prednisone/prednisolone or deflazacort is the standard of care for dystrophinopathies. Improved understanding of real-world switching is needed following deflazacort approval. 
Chart review conducted by 55 neurologists for patients with dystrophinopathy who switched from prednisone/prednisolone to deflazacort between February 2017 and December 2018.  
Charts were reviewed for 102 males in community (49%) and academic (51%) settings. Seventy had Duchenne muscular dystrophy; mean ± SD age 11.6 ± 10.4 years and 80% ambulatory at switch.  Thirty-two had Becker’s muscular dystrophy; mean age 21.2 ± 12.5 years and 81% ambulatory at switch. Average treatment duration was 3.3 years for prednisone/prednisolone and 6 months for deflazacort prior to the chart extraction. Physicians ranked “desire to slow disease progression” and “tolerability issues” as primary reasons for switching in 78% and 57%, respectively. Switching was reported as “very” or “somewhat” effective at addressing primary reasons in 95% of patients.  Commonly recorded adverse effects during prednisone/prednisolone and deflazacort treatment included weight gain (38% and 15%), Cushingoid appearance (26% and 10%), increased appetite (20% and 6%), central obesity (10% and 3%), and fluid retention (9% and 2%); hirsutism occurred for 2% and 4%.  Findings were similar in the subgroup (n=62) with ≥ 3 months deflazacort. Among 34 patients with recorded Global Impression of Illness Severity, 8 (24%) improved, 24 (70%) remained the same, and 2 (6%) worsened after switching.

In this real-world study, the majority of switching from prednisone/prednisolone to deflazacort aimed to improve benefit-risk. During 6-months average follow-up after switching in this uncontrolled study, physicians reported lower proportions of most adverse effects compared to the pre-switch period. Among the minority of charts with disease severity recorded, most showed stability of progressive disease.

Authors/Disclosures

PRESENTER 		No disclosure on file
Claudio Santos No disclosure on file
Brian Pfister Dr. Pfister has received personal compensation for serving as an employee of Praxis Precisions Medicines. Dr. Pfister has stock in Praxis Precision Medicines.
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
Julie A. Parsons, MD Dr. Parsons has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Parsons has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Parsons has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Parsons has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ultragenex. The institution of Dr. Parsons has received research support from AveXis. The institution of Dr. Parsons has received research support from Biogen. The institution of Dr. Parsons has received research support from Scholar Rock. The institution of Dr. Parsons has received research support from Biohaven. The institution of Dr. Parsons has received research support from PTC Therapeutics. The institution of Dr. Parsons has received research support from Novartis.
No disclosure on file