Real-World Osilodrostat Effectiveness and Safety in Nonpituitary Cushing Syndrome
Authors' conclusion
Osilodrostat is a suitable treatment for endogenous CS of various nonpituitary etiologies.
Study context
- Population
- Adults with nonpituitary Cushing syndrome (CS) initiating osilodrostat in France (2019–2022), data ≤36 months52 in mITT
- Participants
- 103 adults
- Exposure
- Ectopic adrenocorticotropin secretion (EAS) vs adrenocortical carcinoma (ACC) vs adrenal adenoma (AA) vs BND
- EASn = 53
- ACCn = 19
- AAn = 17
- BNDn = 14
- Outcome
- Proportion with mean UFC ≤ULN (mITT population)
- Follow-up
- Wk 12
Methodological detailsmulticenter, real-world, 43 patients remained on osilodrostat throughout observation, Osilodrostat initiated and titrated per investigator judgment, Median (min-max) exposure 177 days (1-1178 days); baseline dose 5.0 mg/day (1-60 mg/day)
- Multicenter
- Real-world
- 43 patients remained on osilodrostat throughout observation
- Osilodrostat initiated and titrated per investigator judgment
- Median (min-max) exposure 177 days (1-1178 days); baseline dose 5.0 mg/day (1-60 mg/day)
Main finding
As reported in the abstract
mUFC ≤ULN
Patients with mean urinary free cortisol (mUFC) ≤upper limit of normal (ULN) (mITT; missing = nonresponders)
23 of 52
44.2%; 95% CI, 30.5%–58.7%
Observational data: associations, not effects.
Further findings
| Outcome | EAS | ACC | AA | BND |
|---|---|---|---|---|
| mUFC ≤ULN by etiology (Wk 12 mUFC available, 45 of 52) | 41% | 4/6 events | 1/3 events | 6/7 events |
| Deaths from AEs | 24 events | 5 events | — not stated in the abstract | — not stated in the abstract |
— not stated in the abstract
- Deaths from AEs — Total deaths from AEs
- 29
- Deaths from AEs — Assessed as osilodrostat related
- 1
- Deaths from AEs — Neoplasm progression (most common)
- 11
Cortisol over time
- Cortisol decreased by Wk 4, remaining stable thereafter
Safety
Most common (≥15%) treatment-emergent AEs
- Adrenal insufficiency
- 28%
- Hypokalemia
- 18%
Original publication
Real-World Osilodrostat Effectiveness and Safety in Nonpituitary Cushing Syndrome. J Clin Endocrinol Metab. Published 19 November 2025. doi:10.
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