The impact of deflazacort on puberty in duchenne muscular dystrophy

Background The routine use of glucocorticoids has increased the longevity of patients with Duchenne muscular dystrophy. Long-term steroid therapy may have adverse effects on endocrine function and could influence the onset of puberty. Methods We assessed the pubertal development of our patients who were 14 years of age or older and had been treated with deflazacort as their only glucocorticoid. Results Half (6 of 12) of the boys who were treated with deflazacort had pubertal delay. There was no difference in the age of onset, dose, or duration of deflazacort therapy between those who did and did not have delayed puberty. Conclusions This pilot study suggests that delayed puberty should be studied in future trials that address different doses and schedules of deflazacort therapy in Duchenne muscular dystrophy.