Observational studyThe Journal of clinical endocrinology and metabolism2026
Real-World Osilodrostat Effectiveness and Safety in Nonpituitary Cushing Syndrome.
Observational study in The Journal of clinical endocrinology and metabolism, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05633953 (A Retrospective Observational Study to Evaluate the Safety and Effectiveness of Osilodrostat for the Treatment of Non-Cushing's Disease Cushing's Syndrome), which is not on this map. Cited by 4 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Retrospective Observational Study to Evaluate the Safety and Effectiveness of Osilodrostat for the Treatment of Non-Cushing's Disease Cushing's Syndrome (LINC7)
Who cites it
4 citing papers in PubMed.
- International real-world study on osilodrostat efficacy and safety in adrenal Cushing syndrome.The Journal of clinical endocrinology and metabolism · 2026Article
- Real-World Osilodrostat Effectiveness and Safety in Nonpituitary Cushing Syndrome.The Journal of clinical endocrinology and metabolism · 2026Observational
- Individualized osilodrostat treatment for patients with ACTH-dependent Cushing's syndrome: real-world evidence.Frontiers in endocrinology · 2026Article
- Metyrapone in the Management of Severe Hypercortisolism Secondary to Ectopic Cushing's Syndrome.Case reports in endocrinology · 2026Article
Corrections and comments
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Authors and funding
18 authors.
Funding
Abstract
contextOsilodrostat's clinical development program mostly enrolled Cushing disease patients. Data in nonpituitary Cushing syndrome (CS) patients are limited.
objectiveThis work aims to evaluate osilodrostat effectiveness and safety in nonpituitary CS in real-world practice in France.
methodsA retrospective, observational study (LINC 7; NCT05633953) was conducted in a multicenter institutional practice setting. Data for patients who initiated osilodrostat under the French Autorisation Temporaire d'Utilisation program or, once approved, in routine clinical practice were extracted retrospectively for 36 months or less (2019-2022). Participants included 103 adult nonpituitary CS patients: ectopic adrenocorticotropin secretion (EAS), n = 53; adrenocortical carcinoma (ACC), n = 19; adrenal adenoma (AA), n = 17; and bilateral adrenal nodular disease (BND), n = 14. Forty-three patients remained on osilodrostat throughout the observation period. Median (minimum-maximum) osilodrostat exposure and baseline dose were 177 days (1-1178 days) and 5.0 mg/day (1-60 mg/day), respectively. The main outcome measure was the proportion with mean urinary free cortisol (mUFC) less than or equal to the upper limit of normal (ULN) at Wk 12 (modified intention-to-treat [mITT] population: all enrolled patients with ≥12 weeks' follow-up, excluding patients without Wk 12 mUFC for nonsafety reasons).
resultsOsilodrostat was initiated and titrated based on investigator judgment. Cortisol decreased by Wk 4, remaining stable thereafter. Twenty-three of 52 patients (mITT, 44.2%; 95% CI, 30.5%-58.7%) had mUFC less than or equal to the ULN at Wk 12 (missing values reported as nonresponders). Forty-five of 52 had Wk 12 mUFC available; the proportions with mUFC less than or equal to the ULN by etiology were as follows: EAS, n = 12/29 (41%); ACC, n = 4/6; AA, n = 1/3; and BND, n = 6/7. The most common (≥15%) treatment-emergent adverse events were adrenal insufficiency (28%) and hypokalemia (18%). Twenty-nine patients (EAS, n = 24; ACC, n = 5) died from adverse events (n = 1 assessed as osilodrostat related by investigator), most commonly neoplasm progression (n = 11).
conclusionOsilodrostat is a suitable treatment for endogenous CS of various nonpituitary etiologies.
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