Retrospective analysis to assess clinical impact and side effects of daily versus two consecutive day pulse steroids in DMD boys.   

Corticosteroid therapy is standard of care for boys with Duchenne Muscular Dystrophy (DMD) demonstrating prolonged ambulation and lifespan. Chronic use of daily corticosteroids causes systemic side effects. Higher dose pulse corticosteroids taken on two consecutive days each week have been proposed as a modified treatment regiment to maintain muscle benefits with fewer side effects.

Twenty-four patients were recruited from MDA Clinic at Lurie Children’s (NCT#03319030) who took daily or pulse corticosteroids (prednisone or deflazacort). All patients received clinical standard of care and provided a blood serum sample.

Both cohorts, daily and pulse corticosteroids, had 12 patients with similar fraction of boys taking prednisone (5/12 and 7/12).

Age (p=0.20), height (p=0.21), weight (p=0.37) and treatment duration (54±7 vs 47±7 months, p=0.41) were similar in the daily and pulse groups. Cardiac measurements did not differ between cohorts, i.e. PR interval (p = 0.34), left ventricle septum thickness (p=0.97), fractional shortening (p=0.78). Functional measurements were not significantly different, i.e. 10 meter run test (p=0.79), Brooke’s functional score (p = 0.57) and creatine kinase levels (p = 0.31).

Strikingly, the pulse weekend cohort had improved metabolic and bone health measures compared to the daily steroid cohort.  Whole body fat mass % was reduced: daily = 50.4% vs pulse = 36.3% (p=0.001).  Whole body lean mass % was increased: daily = 48% vs pulse = 64% (p=0.001).  Total body bone mineral density Z-scores were greater: daily = -2.92 vs pulse = -1.20 (p=0.001).  Glucose levels were lower: daily = 126 mg/dL vs pulse = 105 mg/dL (p=0.005).