Endocrinol Metab.  2025 Feb;40(1):26-38. 10.3803/EnM.2024.501.

Medical Treatment of Cushing’s Syndrome

• Guignat L
• Bertherat J

• KMID: 2565248
• DOI: http://doi.org/10.3803/EnM.2024.501

Abstract

Endogenous Cushing’s syndrome (CS) refers to the manifestations of chronic cortisol excess. This rare disease is associated with multiple comorbidities, impaired quality of life, and increased mortality. The management of CS remains challenging. Regardless of the underlying cause, surgical resection of the tumor is typically the first-line and preferred treatment. However, when surgery is not feasible or has been unsuccessful, medical therapies may be employed to control CS. The therapeutic strategy should be individualized based on the recommendations of a multidisciplinary team of experts and the patient’s preferences, informed by detailed information on the available options. All medications require careful monitoring, along with adequate instructions for patients and caregivers. The aim of this mini-review is to provide an overview of the main medical therapies currently used to treat CS, including their efficacy, safety, and management. Despite the availability of new drugs in recent years, the need remains for more effective specific targeted pharmacological therapies.

Keyword

Cushing syndrome; Cushing disease; Cortisol; Steroidogenesis

Figure

• 

  Fig. 1. Sites of action of adrenally directed drugs. CYP11A1, cholesterol side-chain cleavage enzyme; MIT, mitotane; KET, ketoconazole; CYP17A1, cytochrome P450 family 17 subfamily A member 1; DHEA, dehydroepiandrosterone; 3β-HSD2, 3 b-hydroxysteroid dehydrogenase; CYP21A2, cytochrome P450 family 21 subfamily A member 2; CYP11B2, cytochrome P450 family 11 subfamily B member 2; MET, metyrapone; OSI, osilodrostat; CYP11B1, cytochrome P450 family 11 subfamily B member 1.

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