{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,12]],"date-time":"2026-10-12T16:02:15Z","timestamp":1791820935880,"version":"4.3.4"},"reference-count":38,"publisher":"The Endocrine Society","issue":"5","license":[{"start":{"date-parts":[[2025,3,15]],"date-time":"2025-03-15T00:00:00Z","timestamp":1741996800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100017559","name":"Recordati Rare Diseases","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100017559","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100006751","name":"USA","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006751","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,3,24]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:sec>\n                  <jats:title>Context<\/jats:title>\n                  <jats:p>In clinical trials, osilodrostat (11\u03b2-hydroxylase inhibitor) effectively reduced cortisol levels in patients with endogenous Cushing syndrome (CS).<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Objectives<\/jats:title>\n                  <jats:p>A real-world study (ILLUSTRATE) was conducted evaluating osilodrostat use in patients with various etiologies of CS in the United States.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>A retrospective chart-review study was conducted of adults with CS treated with osilodrostat between May 1, 2020, and October 29, 2021.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>A total of 42 patients (Cushing disease, n = 34; CS due to adrenal adenoma, n = 5; ectopic adrenocorticotropin syndrome [EAS], n = 3) were included. Starting doses were 2\u2005mg twice daily in 27\/42 patients (64.3%), maintenance doses were 2\u2005mg twice daily in 6 of 9 patients (66.7%) attaining them. During osilodrostat treatment, urinary free cortisol (UFC) decreased below the upper limit of normal (ULN) in 14 of 20 patients (70.0%) with pretreatment UFC greater than the ULN. Osilodrostat response was observed across a range of doses (2-20\u2005mg\/day). In Cushing disease, median UFC and late-night salivary cortisol decreased from 3.03 and 2.39 \u00d7 ULN, respectively, to 0.71 and 1.13 \u00d7 ULN at last assessment in those with available data (n = 17 and 8, respectively). UFC decreased in all patients with adrenal CS or EAS with available data (n = 2 each). There were no unexpected safety signals; the most common adverse events (incidence \u226520%) were fatigue, nausea, and lower-extremity edema. Glucocorticoid withdrawal syndrome and\/or adrenal insufficiency were reported in 12 of 42 patients (28.6%) after osilodrostat initiation, resulting in treatment discontinuation in 4.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusion<\/jats:title>\n                  <jats:p>In routine practice with dosing individualized according to clinical condition, response, and tolerability, osilodrostat was effective and well tolerated regardless of CS etiology and severity.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1210\/jendso\/bvaf046","type":"journal-article","created":{"date-parts":[[2025,3,15]],"date-time":"2025-03-15T07:40:26Z","timestamp":1742024426000},"source":"Crossref","is-referenced-by-count":15,"title":["Osilodrostat Treatment of Cushing Syndrome in Real-World Clinical Practice: Findings From the ILLUSTRATE study"],"prefix":"10.1210","volume":"9","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9284-6289","authenticated-orcid":false,"given":"Maria","family":"Fleseriu","sequence":"first","affiliation":[{"name":"Departments of Medicine and Neurological Surgery, Pituitary Center, Oregon Health & Science University , Portland, OR 97239 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6815-6181","authenticated-orcid":false,"given":"Richard J","family":"Auchus","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Division of Metabolism, Endocrinology and Diabetes, University of Michigan , Ann Arbor, MI 48109 ,","place":["USA"]},{"name":"Department of Pharmacology, University of Michigan , Ann Arbor, MI 48109 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7329-1887","authenticated-orcid":false,"given":"Wenyu","family":"Huang","sequence":"additional","affiliation":[{"name":"Division of Endocrinology, Metabolism and Molecular Medicine, Northwestern University Feinberg School of Medicine , Chicago, IL 60611 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanna L","family":"Spencer-Segal","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Division of Metabolism, Endocrinology and Diabetes, University of Michigan , Ann Arbor, MI 48109 ,","place":["USA"]},{"name":"Michigan Neuroscience Institute, University of Michigan , Ann Arbor, MI 48109-2800 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kevin C J","family":"Yuen","sequence":"additional","affiliation":[{"name":"Department of Neuroendocrinology and Neurosurgery, Barrow Pituitary Center, Barrow Neurological Institute, St Joseph's Hospital and Medical Center , Phoenix, AZ 85013 ,","place":["USA"]},{"name":"Department of Medicine, University of Arizona College of Medicine and Creighton School of Medicine , Phoenix, AZ 85012 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kelley C","family":"Dacus","sequence":"additional","affiliation":[{"name":"Recordati Rare Diseases Inc , Bridgewater, NJ 08807 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julianne","family":"Padgett","sequence":"additional","affiliation":[{"name":"Recordati Rare Diseases Inc , Bridgewater, NJ 08807 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth K","family":"Babler","sequence":"additional","affiliation":[{"name":"Recordati Rare Diseases Inc , Bridgewater, NJ 08807 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashis K","family":"Das","sequence":"additional","affiliation":[{"name":"PHAR (Partnership for Health Analytic Research) , Beverly Hills, CA 90212 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2277-0068","authenticated-orcid":false,"given":"Cynthia","family":"Campos","sequence":"additional","affiliation":[{"name":"PHAR (Partnership for Health Analytic Research) , Beverly Hills, CA 90212 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael S","family":"Broder","sequence":"additional","affiliation":[{"name":"PHAR (Partnership for Health Analytic Research) , Beverly Hills, CA 90212 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adriana G","family":"Ioachimescu","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Endocrinology and Molecular Medicine, Medical College of Wisconsin , Milwaukee, WI 53226 ,","place":["USA"]},{"name":"Department of Neurosurgery, Medical College of Wisconsin , Milwaukee, WI 53226 ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"80","published-online":{"date-parts":[[2025,3,15]]},"reference":[{"issue":"10","key":"2025041109515142300_bvaf046-B1","doi-asserted-by":"crossref","first-page":"581","DOI":"10.1038\/s41574-023-00868-7","article-title":"An individualized approach to the management of Cushing disease","volume":"19","author":"Fleseriu","year":"2023","journal-title":"Nat Rev Endocrinol"},{"issue":"10418","key":"2025041109515142300_bvaf046-B2","doi-asserted-by":"crossref","first-page":"2237","DOI":"10.1016\/S0140-6736(23)01961-X","article-title":"Cushing's syndrome","volume":"402","author":"Gadelha","year":"2023","journal-title":"Lancet"},{"issue":"8","key":"2025041109515142300_bvaf046-B3","doi-asserted-by":"crossref","first-page":"e13113","DOI":"10.1111\/jne.13113","article-title":"Long-term morbidity and mortality in 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