Medical therapy for Cushing disease is primarily used to control hypercortisolism in patients whose disease persists or with recurrent disease after pituitary surgery, including those awaiting the salutary effects of radiation therapy. In can also be used to control hypercortisolism preoperatively, and in patients who decline surgery or whose tumor location is unknown. Steroidogenesis inhibitors, centrally acting agents, and glucocorticoid receptor antagonists are currently available to treat hypercortisolism, and several novel agents are in development. Given the absence of head-to-head clinical trials, choice between treatments has to be individualized based on careful consideration of patient, tumor, and disease characteristics.
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