Corticosteroid Treatment and Growth Patterns in Ambulatory Males with Duchenne Muscular Dystrophy (P5.082)

Objective/Background: To evaluate growth patterns of ambulatory, steroid-treated males with Duchenne muscular dystrophy (DMD) compared to ambulatory, steroid-naive males with DMD and age-matched unaffected general population males, and to test associations between growth and steroid treatment patterns among steroid-treated males. Design/Methods: Using data from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet), a total of 1,768 height, 2,246 weight and 1,755 BMI measurements between age 2-12 years for 324 ambulatory males, who were treated with steroids for at least 6 months, were identified. Growth curve comparisons and linear mixed-effects modeling, adjusted for race/ethnicity and birth year, were used to evaluate growth and steroid treatment patterns (age at initiation, dosing interval, duration, and cumulative dose). Results: Growth curves for ambulatory, steroid-treated males showed significantly shorter stature, heavier weight, and greater BMI compared to ambulatory, steroid-naive males with DMD and general population US males. Adjusted linear mixed-effects models for ambulatory steroid-treated males showed earlier initiation, daily dosing, longer duration, and greater doses predicted shorter stature for Prednisone. Longer duration and greater doses predicted shorter stature for Deflazacort. At least daily Prednisone dosing predicted lighter weight, but longer duration and greater doses predicted heavier weight. Early initiation, < daily dosing, longer duration and greater doses predicted greater BMIs. Deflazacort predicted shorter stature, but lighter weight, compared to Prednisone. Conclusions: Prolonged steroid use is significantly associated with short stature and heavier weight. Growth alterations associated with steroid treatment should be considered when making treatment decisions for males with DMD. Disclosure: Dr. Lamb has nothing to disclose. Dr. West has nothing to disclose. Dr. Ouyang has nothing to disclose. Dr. Yang has nothing to disclose. Dr. Weitzenkamp has nothing to disclose. Dr. James has nothing to disclose. Dr. Ciafaloni has nothing to disclose. Dr. Pandya has nothing to disclose. Dr. Fox has nothing to disclose. Dr. DiGuiseppi has nothing to disclose.