Corticosteroid Treatments in Males with Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation. Kim, S. et al. Journal of Child Neurology 2015 Vol. 30 (10) 1275-1280.
Submitted by Leigh Ramos-Platt, MD, News Science Editorial Board
In this study by Kim,S. et al, the association between steroid use and loss of ambulation in Duchenne Muscular Dystrophy (DMD) was evaluated in 477 patients followed at multiple sites over 30 years. They were divided into three groups: (1) untreated (2) those treated for 3 months to 3 years, and (3) those that were treated over 3 years – average was 5.4 years of treatment. Regimens including prednisone/prednisolone or deflzacort at standard doses (approximately 0.75 mg/kg/day and 0.9 mg/kg/day respectively) were permitted. Patients who changed regimens (i.e. were on prednisone/prednisolone and deflazacort at different periods) were included.
The investigators found that patients in the long-treatment group were able to ambulate for 2 years more than the untreated patients. Unexpectedly, patients in the short treatment group lost ambulation 0.8 years sooner than those in the untreated group. This finding was thought to be due to combination of more advanced disease and greater side effect profile in the short-treatment group.
Overall, this study indicates that earlier and longer use of steroids is beneficial for patients with DMD. It should be kept in mind that data was collected over 30 years, before steroids were standard of care for patients with DMD.
About the News Science Editorial Board
The board helps to highlight significant, timely science news items for AANEM members. It reviews articles in journals and websites, identifies newsworthy items in the field, and writes article summaries.