Sue Apkon, MD, Chief of Pediatric Rehabilitation Medicine, Children’s Hospital Colorado, discusses a real-world study of patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) who switched from prednisone to deflazacort.
The data was presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific Conference.
As Dr. Apkon explains, the study was a retrospective chart review where 55 neurologists contributed data for 92 male patients with DMD (n=62) or BMD (n=30) who switched from prednisone to deflazacort. Physicians ranked desire to slow disease progression, tolerability, and caregiver or patient request as the most common motivations for switching. Clinical Global Impression (CGI) of Improvement scores were collected at the clinic visit closest to steroid switching during prednisone treatment, and the last available clinic visit during deflazacort treatment. The average treatment duration was 3.3 years for prednisone and 6 months for deflazacort prior to the chart extraction.
Results found that after switching, most patients’ disease progression improved or stabilized. Additionally, switching was reported as “very” or “somewhat” effective at addressing primary concerns in 95% of patients with DMD and 90% of patients with BMD.
To learn more about DMD and BMD, as well as other rare musculoskeletal diseases, visit checkrare.com/diseases/musculoskeletal