{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,28]],"date-time":"2026-07-28T13:12:10Z","timestamp":1785244330736,"version":"3.55.0"},"reference-count":0,"publisher":"The Endocrine Society","license":[{"start":{"date-parts":[[2026,7,28]],"date-time":"2026-07-28T00:00:00Z","timestamp":1785196800000},"content-version":"am","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Context<\/jats:title>\n                    <jats:p>Osilodrostat has demonstrated efficacy in clinical trials for Cushing\u2019s syndrome (CS). Real-world data remain limited.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>To assess real-world effectiveness and safety of osilodrostat in a large cohort of CS patients.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design&amp;Setting<\/jats:title>\n                    <jats:p>Multicenter, retrospective, phase IV-study across 11 Italian endocrine referral centers.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Patients<\/jats:title>\n                    <jats:p>100 CS patients [74% pituitary, 16% adrenal, 10% ectopic].<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Main Outcome Measures<\/jats:title>\n                    <jats:p>Primary endpoint was the percentage of patients with UFC\u2264ULN at their last observation. Secondary endpoints included biochemical and hormonal control, clinical outcomes, QoL, AEs.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                    <jats:p>The mean\u00b1SD (median,range) time to the first normalization of UFC levels, in the 80 patients who achieved normalization, was 92.1\u00b186.3 (67.5,8\u2013455) days, at a mean\u00b1 SD (median,range) osilodrostat dose of 7.2\u00b16.8 (5,1\u201340) mg\/day.<\/jats:p>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>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.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1210\/clinem\/dgag297","type":"journal-article","created":{"date-parts":[[2026,7,27]],"date-time":"2026-07-27T11:22:21Z","timestamp":1785151341000},"source":"Crossref","is-referenced-by-count":0,"title":["Real-World Effectiveness and Safety of Osilodrostat in Cushing\u2019s Syndrome: A Retrospective Phase IV Study"],"prefix":"10.1210","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9632-1348","authenticated-orcid":false,"given":"Rosario","family":"Pivonello","sequence":"first","affiliation":[{"name":"Department of Clinical Medicine and Surgery, Section of Endocrinology, Diabetology, Andrology and Nutrition, University of Naples Federico II , Naples ,","place":["Italy"]},{"name":"UNESCO Chair for Health Education and Sustainable Development, University of Naples Federico II , Naples ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Simeoli","sequence":"additional","affiliation":[{"name":"Department of Clinical Medicine and Surgery, Section of Endocrinology, Diabetology, Andrology and Nutrition, University of Naples Federico II , Naples ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicola","family":"Di Paola","sequence":"additional","affiliation":[{"name":"Department of Clinical Medicine and Surgery, Section of Endocrinology, Diabetology, Andrology and Nutrition, University of Naples Federico II , Naples ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9333-5189","authenticated-orcid":false,"given":"Ilaria","family":"Bonaventura","sequence":"additional","affiliation":[{"name":"Department of Experimental Medicine, Sapienza University of Rome , Rome ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0004-3803-4350","authenticated-orcid":false,"given":"Davide","family":"Ferrari","sequence":"additional","affiliation":[{"name":"Department of Experimental Medicine, Sapienza University of Rome , Rome ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giacomo","family":"Voltan","sequence":"additional","affiliation":[{"name":"Endocrinology Unit, University of Padua , Padua ,","place":["Italy"]},{"name":"Department of Medicine (DIMED), University of Padua; Endocrinology Unit, University Hospital of Padua , Padua ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8713-3532","authenticated-orcid":false,"given":"Roberta","family":"Giordano","sequence":"additional","affiliation":[{"name":"Department of Biological and Clinical Sciences, University of Turin , Turin ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Elena","family":"Aloini","sequence":"additional","affiliation":[{"name":"Endocrinology Unit, Department of Clinical and Molecular Medicine, Sant'Andrea University Hospital, Faculty of Medicine and Psychology, Sapienza University of Rome , Rome ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ylenia","family":"Alessi","sequence":"additional","affiliation":[{"name":"Department of Biomedical, Dental, Morphological and Functional Imaging Sciences, University of Messina , Messina ,","place":["Italy"]},{"name":"Endocrine Unit, University Hospital G. Martino , Messina ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marianna","family":"Martino","sequence":"additional","affiliation":[{"name":"Clinic of Endocrinology and Metabolic Diseases, Azienda Ospedaliero-Universitaria delle Marche, Ancona, \u00a0","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0556-7650","authenticated-orcid":false,"given":"Emanuele","family":"Ferrante","sequence":"additional","affiliation":[{"name":"Endocrinology Unit, IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico , Milan ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federico","family":"Gatto","sequence":"additional","affiliation":[{"name":"Endocrinology Unit, Department of Internal Medicine and Medical Specialties, University of Genoa , Genoa ,","place":["Italy"]},{"name":"IRCCS Ospedale Policlinico San Martino , Genoa ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2883-6139","authenticated-orcid":false,"given":"Soraya","family":"Puglisi","sequence":"additional","affiliation":[{"name":"Internal Medicine Unit, Department of Clinical and Biological Sciences, San Luigi Gonzaga University Hospital, University of Turin , Turin ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vincenzo","family":"Rochira","sequence":"additional","affiliation":[{"name":"Department of Biomedical, Metabolic and Neural Sciences, University of Modena and Reggio Emilia , Modena ,","place":["Italy"]},{"name":"Endocrinology Unit, Department of Medical Specialties, Azienda Ospedaliero-Universitaria of Modena, Baggiovara Hospital , Modena ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Diego","family":"Ferone","sequence":"additional","affiliation":[{"name":"Endocrinology Unit, Department of Internal Medicine and Medical Specialties, University of Genoa , Genoa ,","place":["Italy"]},{"name":"IRCCS Ospedale Policlinico San Martino , Genoa ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9065-3886","authenticated-orcid":false,"given":"Giovanna","family":"Mantovani","sequence":"additional","affiliation":[{"name":"Endocrinology Unit, IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico , Milan ,","place":["Italy"]},{"name":"Department of Clinical Sciences and Community Health, University of Milan , Milan ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6204-3423","authenticated-orcid":false,"given":"Giorgio","family":"Arnaldi","sequence":"additional","affiliation":[{"name":"Unit of Endocrinology, Department of Health Promotion, Mother and Child Care, Internal Medicine and Medical Specialties (PROMISE), Policlinico Paolo Giaccone, University of Palermo , Palermo ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Ferra\u00f9","sequence":"additional","affiliation":[{"name":"Endocrine Unit, University Hospital G. Martino , Messina ,","place":["Italy"]},{"name":"Department of Human Pathology G. 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