What is the role of medical therapy in adrenal-dependent Cushing’s syndrome?
Abstract
Medical therapy to control hypercortisolism in adrenal Cushing's syndrome is currently not the first-line therapy. However, in many clinical scenarios like pre-surgical treatment, in patients who are not suitable candidates for surgery or in patients with bilateral hyperplasia, medical therapy can be important representing the only viable treatment option. Adrenal steroidogenesis inhibitors and glucocorticoid receptor blockers have been used for many years: metyrapone, ketoconazole and mifepristone are in current use and effective. Mitotane can be used as well but is considered second-line therapy because of its high toxicity. Etomidate has a special position as emergency medication in severe hypercortisolism. New drugs are tested in prospective trials (levoketoconazole, osilidrostat and relacorilant) and might become effective alternatives to common drugs. Oher drugs - adrenal steroidogenesis inhibitors as well as glucocorticoid receptor antagonists - are currently tested in vitro.
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Ketoconazole is normally administered in doses between 400-1200 mg/day.
“ketoconazole normally is administrated in doses between 400-1200 mg/day (60)”
From Establishment of a novel human fetal adrenal culture model that supports de novo and manipulated steroidogenesis (2021) · cites this paper as [60]
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Melau, C., Riis, M. L., Nielsen, J. E., Perlman, S., Lundvall, L., Thuesen, L. L., Hare, K. J., Hammerum, M. S., Mitchell, R. T., Frederiksen, H., et al. (2021) · Figshare · doi:10.6084/m9.figshare.16584675
Melau, C., Riis, M. L., Nielsen, J. E., Perlman, S., Lundvall, L., Thuesen, L. L., Hare, K. J., Hammerum, M. S., Mitchell, R. T., Frederiksen, H., et al. (2021) · Figshare · doi:10.6084/m9.figshare.16584672
Melau, C., Riis, M. L., Nielsen, J. E., Perlman, S., Lundvall, L., Thuesen, L. L., Hare, K. J., Hammerum, M. S., Mitchell, R. T., Frederiksen, H., et al. (2021) · Figshare · doi:10.6084/m9.figshare.16584678
Störungen des Cortisol-Haushaltes
Osswald-Kopp, A. (2026) · CME · doi:10.1007/s11298-026-4573-8
- Authors
- Braun, L. T., & Reincke, M.
- Affiliations
- LMU Klinikum (DE)
- Corresponding author
- Braun, Leah T.
- ORCID
- Braun, Leah T.
- Venue
- Best Practice & Research Clinical Endocrinology & Metabolism
- Publisher
- Elsevier BV
- Published
- 2020-05
- Volume
- 34
- Issue
- 3
- Pages
- 101376
- ISSN
- 1521-690X (ISSN-L)
- OpenAlex
- W3003170113
- Cited by
- 21
- References
- 74
- Citations by year
- 2026: 2 · 2025: 6 · 2024: 2 · 2023: 1 · 2022: 3 · 2021: 5 · 2020: 2
- FWCI
- 0.42
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Best Practice & Research Clinical Endocrinology & Metabolism (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Pituitary Gland Disorders and Treatments
- Health Sciences › Medicine › Surgery › Adrenal and Paraganglionic Tumors
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Adrenal Hormones and Disorders
- Keywords
- adrenal Cushing's syndrome, hypercortisolism, medical therapy, metyrapone, ketoconazole, mifepristone, mitotane, etomidate, relacorilant, glucocorticoid receptor antagonist, steroidogenesis inhibitors, bilateral adrenal hyperplasia, drug toxicity
- MeSH terms
- Adrenal Gland Diseases, Cushing Syndrome, Humans, Ketoconazole, Metyrapone, Mitotane, Prospective Studies, Treatment Outcome, Pituitary ACTH Hypersecretion
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