What is it about?
Corticosteroids (steroids) are a well-established, standard treatment for patients with Duchenne muscular dystrophy (DMD), a rare, genetic disease that mainly affects boys. Patients with DMD experience progressive muscle weakness, and few live beyond their third decade of life. Steroids slow disease progression by improving muscle strength and function. However, their side effects can be significant. To ensure that the benefits of treatment outweigh the risks and that the choice of steroid is informed, it is vital to continuously review the long-term experience of patients with DMD treated with steroids. In this article, we review the clinical experience over several years and from many different countries to compare the commonly prescribed steroids, deflazacort and prednisone/prednisolone, with a focus on differences in their mechanism of action, their safety and how effective they are in preserving muscle function. We discovered that, overall, patients receiving deflazacort experience better preservation of muscle function compared with those receiving prednisone/prednisolone. Side effects relating to weight gain and behavior changes appear to be greater with prednisone/prednisolone than with deflazacort, whereas bone health issues, growth delay, and cataracts appear to be more frequent with deflazacort.
Featured Image
Photo by Szilvia Basso on Unsplash
Why is it important?
Healthcare professionals are required to constantly weigh the benefits vs the risks of steroids to ensure their patients with DMD are receiving the best care possible. The choice of steroid, as well as their dose, timing and frequency of use, must therefore be considered carefully based on each patient’s needs. This article summarized what has been learned from previous studies that compare deflazacort and prednisone/prednisolone, the two commonly prescribed steroids. The article will aid healthcare professionals to make informed treatment choice decisions for patients with DMD.
Perspectives
“Clinical studies of patients with DMD present many challenges: in part because of the difficulty of finding well-matched cohorts of patients owing to differences in the age at which they first experience symptoms and the broad spectrum of disease severity. By reviewing data from multiple previous studies that compared deflazacort and prednisone/prednisolone, we have gained a clearer perspective on the drugs’ differences. We found that data from previous studies, which included clinical trials and real-world studies, indicated deflazacort was more effective than prednisone/prednisolone at preserving muscle strength and function in patients with DMD and each steroid was associated with specific side effects.”“Clinical studies of patients with DMD present many challenges: in part because of the difficulty of finding well-matched cohorts of patients owing to differences in the age at which they first experience symptoms and the broad spectrum of disease severity. By reviewing data from multiple previous studies that compared deflazacort and prednisone/prednisolone, we have gained a clearer perspective on the drugs’ differences. We found that data from previous studies, which included clinical trials and real-world studies, indicated deflazacort was more effective than prednisone/prednisolone at preserving muscle strength and function in patients with DMD and each steroid was associated with specific side effects.”
doug biggar
Read the Original
This page is a summary of: Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy, Journal of Neuromuscular Diseases, July 2022, IOS Press,
DOI: 10.3233/jnd-210776.
You can read the full text:
Contributors
The following have contributed to this page
