International real-world study on osilodrostat efficacy and safety in adrenal Cushing syndrome
An erratum to this article was published (J Clin Endocrinol Metab, 15 Jul 2026 ↗)
Authors' conclusion
Osilodrostat controls hypercortisolism in 66.7% of patients with adrenal CS treated for longer than 4 weeks and in 87.5% of cases treated for longer than 12 weeks, with a positive impact on blood pressure and body weight. Patients who received osilodrostat after other previous steroidogenesis inhibitors have a higher probability of response.
Study context
- Population
- Patients with adrenal Cushing syndrome (CS): 16 with ACC and 12 with benign disease; osilodrostat monotherapy in 22, combined with metyrapone in 621 in efficacy evaluation
- Participants
- 28 patients
- Outcome
- Response: Urinary free cortisol (UFC) reduction >50% (complete when UFC below ULN; partial when >50% reduction without normalization)
Methodological detailsinternational, real-world
- International
- Real-world
Main finding
As reported in the abstract
Response (UFC reduction >50%) · treated >4 weeks
| Group | Value | Complete response | Partial response |
|---|---|---|---|
| Osilodrostat | 66.7% | 28.6% | 38.1% |
Osilodrostat
Value66.7%
Complete response28.6%
Partial response38.1%
Observational data: associations, not effects.
Further findings
| Outcome | Values |
|---|---|
| Response in patients treated >12 weeks | Osilodrostat 87.5% |
Predictor of response: osilodrostat as nonfirst-line therapy
- Nonfirst-line use, OR
- 15.0 (P = .010)
Systolic blood pressure and body weight
- Significant decrease in systolic blood pressure and body weight
- P < 0.05
Safety
- Patients with ≥1 adverse event
- 9
- Adverse events leading to osilodrostat discontinuation
- 56% (n = 5)
Original publication
International real-world study on osilodrostat efficacy and safety in adrenal Cushing syndrome. J Clin Endocrinol Metab. Published 13 March 2026. doi:10.
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