Weight and behavior with deflazacort

Deflazacort has a smaller suppressive effect on the hypothalamic-pituitary adrenal (HPA) axis vs prednisone; considering a small concentration of deflazacort crosses the blood brain barrier reaching the HPA axis, a reduced risk of behavioral disorders has been hypothesized.6

Illustration of a human brain in cross-section beside a set of kidneys, representing the HPA axis

Compared to those on prednisone, patients taking deflazacort in clinical trials were7

Less likely to gain weight

Less likely to experience psychiatric side effects (ie, abnormal behavior, aggression, irritability, and mood swings)

Less likely to discontinue treatment due to side effects

  • Psychiatric adverse events generally appeared at a higher rate in the prednisone group vs both deflazacort groups7
    • Abnormal behavior, aggression, irritability, and mood swings were more commonly reported in the prednisone group
    • Psychotic disorder was reported more frequently in the deflazacort group

Study Limitations

Comparisons between deflazacort and prednisone are not included in the Prescribing Information for deflazacort because prednisone is not an approved treatment for DMD.

Real-world Data
Dosing & Administration