Deflazacort vs Prednisone: Comparing Steroid Treatments in Patients with Duchenne Muscular Dystrophy
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.
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.