Real-World Effectiveness and Safety of Osilodrostat in Cushing’s Syndrome: A Retrospective Phase IV Study

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ID: 322893
2026
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Abstract
CONTEXT: Osilodrostat has demonstrated efficacy in clinical trials for Cushing's syndrome (CS). Real-world data remain limited. OBJECTIVE: To assess real-world effectiveness and safety of osilodrostat in a large cohort of CS patients. DESIGN&SETTING: Multicenter, retrospective, phase IV-study across 11 Italian endocrine referral centers. PATIENTS: 100 CS patients [74% pituitary, 16% adrenal, 10% ectopic]. MAIN OUTCOME MEASURES: Primary endpoint was the percentage of patients with UFC≤ULN at their last observation. Secondary endpoints included biochemical and hormonal control, clinical outcomes, QoL, AEs. RESULTS: 81% of patients had normal UFC levels at last observation, 92% at least once within study period. Excluding 12 patients with normal baseline UFC levels, 78.4% of patients had normal UFC levels at their last observation, whereas 90.9% at least once within the study period.The mean±SD (median,range) time to the first normalization of UFC levels, in the 80 patients who achieved normalization, was 92.1±86.3 (67.5,8-455) days, at a mean± SD (median,range) osilodrostat dose of 7.2±6.8 (5,1-40) mg/day.Significant reductions in late-night salivary cortisol and morning serum cortisol levels were observed. Improvements were noted in anthropometric parameters, blood pressure, glucose metabolism, clinical signs, and QoL. AEs occurred in 29% of patients, mostly mild-to-moderate; adrenal insufficiency was the most common AE reported in 20% of patients. Hormonal control and safety profiles were consistent across CS etiologies. CONCLUSIONS: Osilodrostat is effective, generally well tolerated in routine clinical practice, representing a valuable therapy for CS management, able to achieve rapid and sustained hormonal control, significant metabolic, cardiovascular, clinical and QoL improvements with a well-tolerated safety profile.
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Authors Rosario Pivonello, Chiara Simeoli, Nicola Di Paola, Ilaria Bonaventura, Davide Ferrari, Giacomo Voltan, Roberta Giordano, Maria Elena Aloini, Ylenia Alessi, Marianna De Martino, Emanuele Ferrante, Federico Gatto, Soraya Puglisi, Vincenzo Rochira, Diego Ferone, Giovanna Mantovani, Giorgio Arnaldi, Francesco Ferraù, Antonio Stigliano, Emanuela Arvat, Mattia Barbot, Carla Scaroni, Andrea M Isidori, Annamaria Colao
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Journal the journal of clinical endocrinology & metabolism
Year 2026
DOI
10.1210/clinem/dgag297
URL
https://doi.org/10.1210/clinem/dgag297
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