Osilodrostat is a novel potent oral steroidogenesis inhibitor with a non-steroidal chemical structure, recently approved for the treatment of adult patients with endogenous Cushing's syndrome, and Cushing's disease not cured bytab pituitary surgery or in whom pituitary surgery is not an option. Osilodrostat has been evaluated in different multicentre phase II and III clinical studies, and has shown to have notable effects, such as significant reductions in cortisol secretion, associated with significant improvement in body weight, blood pressure, glucose metabolism, lipid profile, psychological status and quality of life. The favourable safety profile, combined with the relevant efficacy, could make osilodrostat suitable as medical treatment in several phases of the Cushing's syndrome treatment journey: before surgery, as preoperative treatment, or instead of surgery, in cases where surgery is not an option or refused, as first-line treatment; after surgery, in cases of persistent or recurrent disease, as second-line treatment; after second surgery or radiotherapy following pituitary surgery as bridging treatment waiting for the definitive disease control, as third-line treatment. Further real-world clinical experience data are needed to confirm the current knowledge.
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http://dx.doi.org/10.17925/EE.2024.20.1.8 | DOI Listing |
J Clin Endocrinol Metab
January 2025
Institut Cochin, INSERM U1016, CNRS UMR8104, Paris Descartes University, Paris, France.
Purpose: Primary bilateral macronodular adrenal hyperplasia (PBMAH), the most common cause of Cushing's syndrome due to bilateral nodules, is a heterogeneous disease at the clinical, hormonal and morphological levels. ARMC5 inactivating pathogenic variants are causative of PBMAH and rare variants of PDE11A have been associated with PBMAH.
Methods: Leukocyte DNA of 354 PBMAH index cases was sequenced for ARMC5 and PDE11A genes by Next generation sequencing (NGS).
Front Endocrinol (Lausanne)
January 2025
Division of Diabetes and Endocrinology, Department of Internal Medicine, Kobe University Hospital, Kobe, Japan.
Metyrapone is commonly used in the initial management of Cushing's syndrome to reduce hypercortisolemia, but its optimal dosage and timing can vary significantly between patients. Currently, there are limited guidelines on adjustment methods for its administration to individual needs. This study aimed to evaluate responsiveness of each patient to metyrapone and identify the patient characteristics associated with the indices of cortisol responsiveness following a low-dose metyrapone.
View Article and Find Full Text PDFExpert Rev Endocrinol Metab
January 2025
Carrera de Medicina Humana, Universidad Científica del Sur, Lima, Perú.
Introduction: Endocrine paraneoplastic syndromes (ePNS) are caused by malignant cells that induce hormonal alterations unrelated to the tissue of origin of the neoplasm. The aim of this manuscript is to review the pathophysiology, diagnosis, and treatment of endocrine paraneoplastic syndromes (ePNS).
Areas Covered: We searched the PubMed/Medline, Embase, and Scielo databases, including 96 articles.
Curr Cardiol Rev
January 2025
Division of Applied Biomedical Science and Biotechnology, School of Health Sciences, IMU University, 126, Jalan Jalil Perkasa 19, Bukit Jalil, 57000 Kuala Lumpur, Malaysia.
Cardiovascular Disease [CVD], the leading cause of death globally, poses a significant burden on the healthcare sector. Its association with stress and Cushing's Syndrome has driven cortisol, the 'stress hormone,' to be a potential candidate in determining CVD risk. Cortisol synthesis and release through the hypothalamic-pituitary-adrenal [HPA] axis are regulated by several hormones and receptors involved in the pathological cascade towards CVD.
View Article and Find Full Text PDFFront Endocrinol (Lausanne)
January 2025
Department of Internal Medicine and Endocrinology, University Clinical Centre of the Medical University of Warsaw, Warsaw, Poland.
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