ArticleThe Journal of clinical endocrinology and metabolism2023
Efficacy and Safety of Osilodrostat in Paraneoplastic Cushing Syndrome: A Real-World Multicenter Study in France.
Article in The Journal of clinical endocrinology and metabolism, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers, 1 of them a synthesis that pooled it.
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Who cites it
47 citing papers in PubMed, 1 synthesis or guideline pooled it, 60 citations in OpenAlex.
- Management of ectopic Cushing syndrome in medullary thyroid carcinoma: from case reports to systematic literature review.Frontiers in endocrinology · 2026Pooled it
- Antineoplastic effect of block-and-replace strategy for managing hypercortisolism caused by adrenal cortical carcinoma.JCEM case reports · 2026Article
- Ectopic ACTH-dependent Cushing syndrome in 3 hospitalized patients: lessons learned from management with osilodrostat.JCEM case reports · 2026Article
- Efficacy and safety of osilodrostat in patients with ectopic Cushing's syndrome.Hormones (Athens, Greece) · 2026Review
- International real-world study on osilodrostat efficacy and safety in adrenal Cushing syndrome.The Journal of clinical endocrinology and metabolism · 2026Article
- Aligning treatment to physiology: when to use chronotherapy, block and replace, or titration to treat hypercortisolism.Journal of the Endocrine Society · 2026Article
- Update and Practical Recommendations for the Use of Medical Treatment of Cushing Syndrome.Endocrine reviews · 2026Review
- Real-World Osilodrostat Effectiveness and Safety in Nonpituitary Cushing Syndrome.The Journal of clinical endocrinology and metabolism · 2026Observational
- Use of PRRT and osilodrostat for ectopic Cushing syndrome secondary to thymic neuroendocrine neoplasm.Endocrinology, diabetes & metabolism case reports · 2026Article
- European Society of Neuroendocrine Tumors (ENETS) 2025 guidance paper for lung and thymic carcinoids.Journal of neuroendocrinology · 2026Article
- Use of osilodrostat in a block-and-replace and titration strategy for cushing's syndrome: a position paper by French experts.Reviews in endocrine & metabolic disorders · 2026Article
- Efficacy and safety of osilodrostat in patients with ectopic Cushing´s syndrome. a real-world study in Spain.Journal of endocrinological investigation · 2026Article
- Alternating Therapy With Osilodrostat and Etomidate in Severe Ectopic Cushing's Syndrome Complicated by Silent Bowel Perforation.Case reports in endocrinology · 2026Article
- Individualized osilodrostat treatment for patients with ACTH-dependent Cushing's syndrome: real-world evidence.Frontiers in endocrinology · 2026Article
- Severe Cushing Syndrome From Ectopic Adrenocorticotropin Secretion In Metastatic Prostate Cancer Treated With Osilodrostat.JCEM case reports · 2025Article
- Blood steroid hormone profile and clinical outcomes following switching from metyrapone to osilodrostat in patients with Cushing disease.Endocrine journal · 2025Article
- Chronotherapy With Once-Daily Osilodrostat Improves Cortisol Rhythm, Quality of Life, and Sleep in Cushing's Syndrome.The Journal of clinical endocrinology and metabolism · 2025Article
- Safety management in Cushing syndrome during osilodrostat treatment based on morning blood cortisol level.Endocrine journal · 2025Article
- Review
- Lung Carcinoids in Adolescents and Young Adults (AYAs): A Still Overlooked Clinical Entity.Current oncology (Toronto, Ont.) · 2025Review
Corrections and comments
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Authors and funding
23 authors at 12 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
contextProspective studies have demonstrated the efficacy of osilodrostat in Cushing disease. No study has evaluated osilodrostat in a series of patients with paraneoplastic Cushing syndrome/ectopic adrenocorticotropin syndrome (PNCS/EAS).
objectiveThis work aimed to evaluate in France the real-world efficacy and safety of osilodrostat in patients with PNCS/EAS.
methodsA total of 33 patients with PNCS/EAS with intense/severe hypercortisolism were involved in this retrospective, multicenter, real-world study. Patients received osilodrostat between May 2019 and March 2022 at a median initial dose (range) of 4 mg/day (1-60) and maximum dose, 20 mg/day (4-100), first under patient then cohort temporary authorizations and after marketing authorization. Regimens used titration (n = 6), block and replace (n = 16), or titration followed by block and replace (n = 11).
resultsIn 11 patients receiving osilodrostat as first-line monotherapy, median 24-hour urinary free cortisol (24h-UFC) decreased dramatically (from 26 × upper limit of normal [ULN; 2.9-659] to 0.11 × ULN [0.08-14.9]; P < .001). In 9 of them, 24h-UFC normalization was achieved in 2 weeks (median). Thirteen additional patients were previously treated with classic steroidogenesis inhibitors but 10 of these 13 were not controlled. In these patients, osilodrostat monotherapy, used as second line, induced a significantly decreased of 24h-UFC (from 2.6 × ULN [1.1-144] to 0.22 × ULN [0.12-0.66]; P < .01). Nine additional patients received osilodrostat in combination with another anticortisolic drug, decreasing 24h-UFC from 11.8 × ULN (0.3-247) to 0.43 × ULN (0.33-2.4) (P < .01). In parallel, major clinical symptoms/comorbidities improved dramatically with improvement in blood pressure, hyperglycemia, and hypokalemia, allowing the discontinuation or dose reduction of patient treatments. Adrenal insufficiency (grade 3-4) was reported in 8 of 33 patients.
conclusionOsilodrostat is a rapidly efficient therapy for PNCS/EAS with severe/intense hypercortisolism. Osilodrostat was generally well tolerated; adrenal insufficiency was the main side effect.
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