Daily Glucocorticoid Therapy Endorsed for Young Duchenne MD Patients

Long-term therapy with daily glucocorticoid therapy can improve clinical outcomes of young patients with Duchenne muscular dystrophy (DMD) without serious side effects, according to recent research.

In “Long-Term Outcome of Interdisciplinary Management of Patients with Duchenne Muscular Dystrophy Receiving Daily Glucocorticoid Treatment,” researchers performed a retrospective study with 97 DMD patients (ages 10 to 16) who had been treated with daily glucocorticoid for a mean of 8.5 years. The majority of the patients (89%) received the glucocorticoid deflazacort (Calcort).

The researchers’ work was published in The Journal of Pediatrics.

Clinical outcomes that were assessed included motor, pulmonary, and cardiac function, as well as scoliosis (abnormal lateral curvature of the spine). Side effects of the therapy included growth and weight gain; facial fullness; hypertrichosis (abnormal amount of hair growth over the body); cataracts; and blood pressure, bone health, gastrointestinal, and behavioral changes.

In patients who were 13 to 16 years old, researchers noted a significant improvement in motor function, with 40% being able to rise from the floor and 50% performing a 30-foot run test. This is particularly important because DMD patients not treated with glucocorticoids generally lose the ability to walk from place to place by age 13. Pulmonary function, measured as forced vital capacity, was well preserved in all the patients.

Researchers detected a change in cardiac function — specifically left ventricle systolic dysfunction — in 13% and 21% of younger (10 to 13 years old) and older patients, respectively. Scoliosis was detected in 6% of the patients (all 16 years old).

Other trends observed were that 86% of patients showed normal weight gain; 30% did not experience an increase in facial fullness; 72% had short stature; and 19% had asymptomatic cataracts. Asymptomatic spine compression deformities were detected in 76%, and there were long bone fractures in 30%.

Overall, the analysis suggests that long-term daily glucocorticoid therapy can result in favorable clinical outcomes for young DMD patients.

“We recommend use of daily glucocorticoid therapy for patients with DMD with anticipatory management of side effects and a coordinated interdisciplinary care approach,” the researchers wrote.