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Management of Osilodrostat Therapy in Patients With Cushing's Syndrome: A Modified Delphi Consensus Panel
Susan L Samson1, Diane Donegan2, Eliza B Geer3
1Departments of Medicine and Neurologic Surgery, Mayo Clinic, Jacksonville, FL 32224, USA.
Journal of the Endocrine Society
|July 7, 2025
Summary
Expert endocrinologists developed guidance for using osilodrostat to treat Cushing's syndrome (CS). This consensus provides recommendations for optimal patient preparation, dosing, and management of osilodrostat therapy in clinical practice.
Area of Science:
- Endocrinology
- Pharmacology
Background:
- Endogenous Cushing's syndrome (CS) involves chronic exposure to excess cortisol.
- Surgery is the primary treatment, but medical therapies like osilodrostat are crucial for non-surgical candidates or persistent disease.
- Osilodrostat, an adrenal steroidogenesis inhibitor, is approved for CS, but individualized treatment guidance is needed.
Purpose of the Study:
- To establish expert-driven recommendations for the optimal use of osilodrostat in managing endogenous Cushing's syndrome.
- To address knowledge gaps regarding the individualization of osilodrostat treatment in various clinical scenarios and patient populations.
Main Methods:
- A modified Delphi consensus panel study involving 13 specialists from high-volume endocrinology centers.
- Three consensus rounds, including two anonymous surveys and a virtual workshop, conducted over 10 months.
Main Results:
- Consensus was reached on 26 statements regarding osilodrostat use.
- Key topics covered patient preparation, baseline testing, dosing strategies, dose adjustments, monitoring, and managing clinical events.
Conclusions:
- The Delphi method yielded expert treatment guidance and recommendations for osilodrostat.
- These recommendations aim to educate physicians and optimize osilodrostat therapy for patients with Cushing's syndrome.
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