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

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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Journal
The Journal of Clinical Endocrinology & Metabolism
Published
2026-07-27
DOI
https://doi.org/10.1210/clinem/dgag297
Primary Topic
Pituitary Gland Disorders and Treatments
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article
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article

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

Emanuela Arvat, Roberta Giordano, Mattia Barbot, Ilaria Bonaventura et al.
The Journal of Clinical Endocrinology & Metabolism
Pituitary Gland Disorders and Treatments
article

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

Emanuela Arvat, Roberta Giordano, Mattia Barbot, Ilaria Bonaventura, Antonio Stigliano, Giorgio Arnaldi, Marianna De Martino, Nicola Di Paola, Giovanna Mantovani, Soraya Puglisi, Rosario Pivonello, Emanuele Ferrante, Giacomo Voltan, Annamaria Colao, Federico Gatto, Vincenzo Rochira, Ylenia Alessi, Maria Elena Aloini, Davide Ferrari, Carla Maria Scaroni, Chiara Simeoli, Francesco Ferraù, Andrea M Isidori, Diego Ferone
article en

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.

The Journal of Clinical Endocrinology & Metabolism
University of Messina (IT), University of Modena and Reggio Emilia (IT), University of Padua (IT), University of Milan (IT), Policlinico Umberto I (IT), Martin University (US), Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT), Azienda Unita' Sanitaria Locale Di Modena (IT), Azienda Ospedaliero Universitaria Ospedali Riuniti (IT), Azienda Ospedaliero-Universitaria di Modena (IT), Federico II University Hospital (IT), Azienda Ospedaliera Sant'Andrea (IT), Department of Medical Sciences (BY), Ospedale Policlinico San Martino (IT), Azienda Ospedaliera Universitaria Policlinico "Paolo Giaccone" di Palermo (IT), Ospedale San Luigi Gonzaga (IT), University of Turin (IT), University of Naples Federico II (IT), University of Genoa (IT), Sapienza University of Rome (IT)
Good health and well-being
Openalex Percentile: Top 8%
Pituitary Gland Disorders and Treatments
DOIOpen Access PDFOpenAlex
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