“Corticosteroids will likely remain the mainstay of treatment for DMD for the foreseeable future and worldwide, so it is important that we establish a scientific evidence-based standard of care,” said Griggs. learn. “This study suggests that health concerns about daily corticosteroid use are overstated and that there is a clear benefit in terms of improving lung and motor function. These findings clearly support this. for daily rather than intermittent regimen as an initial treatment for boys with DMD.”
Treatment of Duchenne muscular dystrophy (DMD)
DMD is a condition that occurs almost exclusively in boys and is characterized by muscle weakness, which appears at age 3-4 and progresses rapidly to lead to severe disability. Symptoms eventually spread to the heart and muscles responsible for breathing, and the disease is often fatal by the time the boy reaches adolescence. An estimated 28,000 people in the US have the disease.
A global survey of physicians treating DMD found 29 different regimens, with the most common being ten days off and ten days off. This intermittent dosing regimen was introduced in an attempt to limit the potential side effects associated with prolonged steroid use in children, such as weight gain, stunted growth, and loss of bone density.
The Finding Optimal Corticosteroid regimen for DMD (FOR-DMD) study was launched in 2013 to compare daily and intermittent steroid use and establish the most beneficial regimen for DMD patients. from the perspective of clinical benefit and safety. Griggs and Kate Bushby, MD, of the University of Newcastle, have begun a phase 3 clinical trial conducted through the Muscle Research Group, an international network of researchers in muscular dystrophy that Griggs has worked with. help was founded in 1997 to advance clinical research into neuromuscular disorders, including DMD.
The new study recruited 196 boys with DMD at 32 study sites across North America and Europe and followed them for three years. Participants were assigned to three groups consisting of a daily regimen of prednisone or deflazacort, or intermittent prednisone, and followed up for three years.
The researchers found that daily regimens of both drugs significantly slowed disease progression as measured by testing of muscle strength and function, compared with the intermittently administered group. Although the daily regimen increases side effects in general, there are very few serious side effects.