Researchers found that giving Duchenne muscular dystrophy (DMD) patients glucocorticoid therapy keeps them mobile for a longer period of time and reduces their risk of dying.
DMD remains incurable, but recent advances have made it possible for patients to live beyond their teens and sometimes into their 50s.
Glucocorticoids, powerful anti-inflammatory compounds, are standard of care for DMD patients. In fact, the U.S. Food and Drug Administration recently approved Emflaza (deflazacort), a glucocorticoid, to treat the disease.
Nonetheless, due to the lack of data on the long-term effects of this class of drugs and its known side effects (for example, increase in blood sugar levels and suppression of the immune system), some physicians wait to prescribe glucocorticoids until patients’ mobility is quite poor.
“Everyone had the idea that long-term use could be beneficial, but nobody had really rigorously tested that,” Heather Gordish-Dressman, a statistician at the Center for Genetic Medicine Research at Children’s National Health System, said in a news release.
Researchers at Children’s National partnered with the Cooperative International Neuromuscular Research Group (CINRG) and designed a prospective study (NCT00468832) to examine glucocorticoids’ long-term effects on the life expectancy and survival of DMD patients. This constituted the largest long-term assessment of people with DMD.
For this study, which involved 20 centers in nine countries, the team enrolled 440 DMD males aged 2 to 28 and followed them for 10 years.
Researchers compared patients who had received glucocorticoid treatment for one year or longer to others who received it for a smaller period of time or didn’t receive glucocorticoid therapy at all.
Researchers assessed the effect of the type of therapy DMD patients were given by evaluating the progression of DMD-related mobility and upper limb milestones: time to stand from a lying down position (supine), and loss of standing ability from supine, four-stair climb, walk, full overhead reach, hand-to-mouth plus hand function.
Scientists found that glucocorticoid treatment for one year or more significantly slowed disease progression compared to patients who received medication for less than a month or not at all. As a result, long-term glucocorticoid receivers maintained their ability to independently change from a lying down to a standing position, climb four stairs or even walk, for a longer period of time.
Also, DMD patients’ average age at loss of upper limb mobility was delayed by 2.3 to 8.0 years in the long-term treated group compared to the others. Moreover, deflazacort increased the median age at loss of three milestones by almost three years when compared to another glucocorticoid commonly prescribed for DMD, prednisone.
“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, said Dr. Craig McDonald, a University of California-Davis professor and lead author of the study. “Most importantly, patients with Duchenne using glucocorticoids experienced an overall reduction in risk of death by more than 50 percent.”