The purpose was to review the use of
mifepristone in the treatment of
Cushing's syndrome (CS) in the context of other recently published studies. We review the use of
mifepristone, as published in the recent Study of the Efficacy and Safety of
Mifepristone in the Treatment of Endogenous
Cushing's Syndrome (SEISMIC). We also review the multiple case reports and case series of
mifepristone use in CS. A review of other medications used in the treatment of
Cushing's disease (CD), including
pasireotide and
cabergoline also provides context for the discussion of the role of
mifepristone in the treatment of CD. The results show that the treatment of CD has been primarily surgical with medical
therapy reserved for adjuvant
therapy when primary treatment fails or other
therapies require time for optimal efficacy. Two recent large prospective studies, using
pasireotide and
mifepristone provide new clinical insights to the medical treatment of CD in particular.
Mifepristone has been used to treat excessive
cortisol production by blocking the action of
cortisol at the level of the
glucocorticoid receptor. Until recently, the majority of clinical experience with
mifepristone on the treatment of excess
cortisol was derived from case reports and small case series. Based on the SEISMIC study,
mifepristone was FDA approved for
hyperglycemia associated with CS. In conclusion the role of
mifepristone in the treatment of CD remains one of adjuvant
therapy. Its place among other choices for medical
therapy has yet to be firmly established and an evidenced-based approach toward the use of novel medications in the treatment of CD has not been made. Selection of medication depends on drug approval and availability in individual countries and requires cautious assessment of potential adverse effects, consideration of patient comorbidities, and efficacy.