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Review
. 2022 Dec;25(6):795-809.
doi: 10.1007/s11102-022-01268-2. Epub 2022 Aug 24.

Treatment of Cushing's syndrome with osilodrostat: practical applications of recent studies with case examples

Maria Fleseriu  1 , Beverly M K Biller  2
Affiliations
Review

Treatment of Cushing's syndrome with osilodrostat: practical applications of recent studies with case examples

Maria Fleseriu et al. Pituitary. 2022 Dec.

Abstract

Endogenous Cushing's syndrome (CS) is a rare endocrine condition frequently caused by a tumor resulting in elevated cortisol levels. Cushing's disease (CD) caused by an adrenocorticotropic hormone-secreting pituitary adenoma is the most common form of endogenous CS. Medical therapy for CD is mostly used as second-line treatment after failed surgery or recurrence and comprises several pituitary-directed drugs, adrenal steroidogenesis inhibitors, and a glucocorticoid receptor blocker, some of which are US Food and Drug Administration (FDA)-approved for this condition. The recent Pituitary Society consensus guidelines for diagnosis and management of CD described osilodrostat, an oral inhibitor of 11β-hydroxylase, as an effective, FDA-approved medical therapy for CD. Because clinical experience outside clinical trials is limited, we provide here a review of published data about osilodrostat and offer example case studies demonstrating practical considerations on the use of this medication. Recommendations regarding osilodrostat are provided for the following situations: specific assessments needed before treatment initiation; monitoring for adrenal insufficiency, hypokalemia, and changes in QTc; the potential value of a slow up-titration in patients with mild disease; managing temporary treatment cessation for patients with CD who have acquired coronavirus disease 2019; monitoring for increased testosterone levels in women; exercising caution with concomitant medication use; considering whether a higher dose at nighttime might be beneficial; and managing cortisol excess in ectopic and adrenal CS. This review highlights key clinical situations that physicians may encounter when using osilodrostat and provides practical recommendations for optimal patient care when treating CS, with a focus on CD.

Keywords: Adrenal steroidogenesis inhibitors; Cushing’s disease; Cushing’s syndrome; Medical therapy; Osilodrostat; Pituitary adenoma.

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Conflict of interest statement

MF reports serving as an investigator with research grants to Oregon Health and Science University from Millendo Therapeutics, Novartis, Recordati, and Strongbridge Biopharma; has received occasional scientific consulting honoraria from HRA Pharma, Novartis, Recordati, Sparrow Pharmaceuticals, and Strongbridge Biopharma; served as a member of the LINC 3 steering committee; and is a member of the editorial board for Pituitary. BMKB reports research grants to her institution from Millendo Therapeutics, Novartis, and Strongbridge Biopharma; has received occasional consulting honoraria from HRA Pharma, Recordati, Sparrow Pharmaceuticals, and Strongbridge Biopharma; and served on the LINC 3 steering committee.

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