Medical therapy of endogenous Cushing's syndrome with steroidogenesis inhibitors: treatment rationale, available drugs, and therapeutic effects.

Endogenous Cushing's syndrome (CS) is a rare disease characterized by a glucocorticoid excess. If inadequately treated, the latter can lead to increased morbidity and mortality. Surgical removal of the underlying tumor is the first line treatment but is sometimes not feasible or even contraindicated. Additionally, in cases with severe CS, rapid control of hypercortisolism may be required. In these scenarios steroidogenesis inhibitors represent a therapeutic alternative to surgery. Over the last years, the knowledge on the broad therapeutic effects of steroidogenesis inhibitors and the number of available drugs have increased. However, large comparative studies are still lacking. Accordingly, the decision on which drug to be used in a certain patient or clinical setting may be difficult. The aim of this review is to summarize the main characteristics of steroidogenesis inhibitors.

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Déclaration de conflit d'intérêts

MD and BA have nothing to declare. TD received travel costs from Recordati Rare Diseases and honoraria for scientific board activities from HRA Pharma. Furthermore, he served a principal investigator and national study coordinator of clinical studies from Corcept Pharmeceuticals.