Glucocorticoids, a class of steroid hormone medications often prescribed to patients with Duchenne muscular dystrophy (DMD), offer long-term benefits for this disease, including longer preservation of muscle strength and function and decreased risk of death. These findings support the standard prescribing practices in many clinics and could help sway parents who are on the fence about their children receiving these therapies, say authors of a study published online Nov. 22, 2017 in The Lancet.
DMD is characterized by loss of muscle function and progressive muscle weakness that begins in the lower limbs and typically affects males due to the location of its causative genetic mutation. Patients with this devastating neuromuscular disease often receive glucocorticoids at some point as the disease progresses. Studies since the late 1980s have confirmed short-term benefits to treating with these drugs, including delaying the loss of muscle strength and function.
However, no prospective study had followed long-term glucocorticoid use in these patients, explains Heather Gordish-Dressman, Ph.D., a statistician at the Center for Genetic Medicine Research at Children’s National Health System and study senior author. The lack of long-term data led some physicians to delay treatment with these drugs since their use can lead to significant side effects, including weight gain, delayed growth and immunosuppression.
“Everyone had the idea that long-term use could be beneficial, but nobody had really rigorously tested that,” Gordish-Dressman says.
Craig McDonald, M.D., a University of California, Davis, professor and lead author of the study adds: “This long-term, follow-up study provides the most definitive evidence that the benefits of glucocorticoid steroid therapy in DMD extend over the entire lifespan. Most importantly, patients with Duchenne using glucocorticoids experienced an overall reduction in risk of death by more than 50 percent.”