Department of Pediatrics
Congenital, Hereditary, and Neonatal Diseases and Abnormalities | Health Services Administration | Health Services Research | Musculoskeletal Diseases | Nervous System Diseases | Pediatrics | Therapeutics
Aim: To assess outcomes among patients with Duchenne muscular dystrophy receiving deflazacort or prednisone in real-world practice.
Methods: Clinical data for 435 boys with Duchenne muscular dystrophy from Cincinnati Children's Hospital Medical Center were studied retrospectively using time-to-event and regression analyses.
Results: Median ages at loss of ambulation were 15.6 and 13.5 years among deflazacort- and prednisone-initiated patients, respectively. Deflazacort was also associated with a lower risk of scoliosis and better ambulatory function, greater % lean body mass, shorter stature and lower weight, after adjusting for age and steroid duration. No differences were observed in whole body bone mineral density or left ventricular ejection fraction.
Conclusion: This single center study adds to the real-world evidence associating deflazacort with improved clinical outcomes.
Duchenne muscular dystrophy, deflazacort, prednisone, real-world data
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© 2020 Analysis Group, Inc. Open access: This work is licensed under the Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license,visit http://creativecommons.org/licenses/by-nc-nd/4.0/
DOI of Published Version
Marden JR, Freimark J, Yao Z, Signorovitch J, Tian C, Wong BL. Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center. J Comp Eff Res. 2020 Feb;9(3):177-189. doi: 10.2217/cer-2019-0170. Epub 2020 Jan 10. PMID: 31922454. Link to article on publisher's site
Journal of comparative effectiveness research
Marden JR, Freimark J, Yao Z, Signorovitch J, Tian C, Wong BL. (2020). Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center. Open Access Publications by UMMS Authors. https://doi.org/10.2217/cer-2019-0170. Retrieved from https://escholarship.umassmed.edu/oapubs/4123
Creative Commons License
This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.
Congenital, Hereditary, and Neonatal Diseases and Abnormalities Commons, Health Services Administration Commons, Health Services Research Commons, Musculoskeletal Diseases Commons, Nervous System Diseases Commons, Pediatrics Commons, Therapeutics Commons