Daily corticosteroid use is significantly more effective than an intermittent regimen for improving motor function in patients with Duchenne muscular dystrophy (DMD), a new study suggests.
Dr Michela Guglieri
"The intermittent regimen has been used to minimize side effects such as weight gain, and what this study shows is that intermittent prednisone has a very similar effect on weight as the daily regimen," study investigator Michela Guglieri, MD, senior clinical lecturer, John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom, told Medscape Medical News.
"On the other hand, intermittent prednisone allowed the children to grow much more than with the daily regimen."
The findings were presented at the American Academy of Neurology (AAN) 2022 Annual Meeting.
Optimal Treatment Unknown
DMD is a genetic disorder resulting in near absence of the dystrophic protein in muscles. Early signs of progressive muscular weakness may include delayed ability to sit, stand, or walk, and difficulties learning to speak.
While there's no known cure for DMD, treatments such as gene therapy can control symptoms in some patients. Research shows corticosteroids improve muscle strength and function in boys with DMD with guidelines recommending them as an option for all newly diagnosed patients.
However, there has been uncertainty about the optimal steroid regimen, which has led to great variability in their use, said Guglieri.
