Osilodrostat: A Novel Potent Inhibitor of 11-Beta-Hydroxylase for the Treatment of Cushing's Syndrome.

touchREV Endocrinol

Dipartimento di Medicina Clinicae Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologiae Nutrizione, Università "Federico II" di Napoli, Naples, Italy.

Published: April 2024

AI Article Synopsis

  • Osilodrostat is a new oral medication approved for treating adults with Cushing's syndrome, particularly for those who haven't had success with surgery.
  • Clinical studies have shown it effectively lowers cortisol levels and improves various health aspects like body weight, blood pressure, and overall quality of life.
  • It has a favorable safety profile and could be used at various stages of Cushing's syndrome treatment, including pre-surgery, post-surgery, and as a first-line or second-line treatment option.

Article Abstract

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.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11132648PMC
http://dx.doi.org/10.17925/EE.2024.20.1.8DOI Listing

Publication Analysis

Top Keywords

cushing's syndrome
12
pituitary surgery
12
treatment
9
osilodrostat novel
8
novel potent
8
surgery
8
surgery option
8
treatment surgery
8
surgery cases
8
osilodrostat
4

Similar Publications

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).

View Article and Find Full Text PDF

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 PDF

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.

View Article and Find Full Text PDF

Serum Cortisol and Cardiovascular Disease Risk-A Potential Biomarker.

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 PDF
Article Synopsis
  • Non-neoplastic hypercortisolaemia, or pseudo-Cushing's syndrome (PCS), can occur due to various health conditions like depression, obesity, and diabetes, linked to overactivity of the hypothalamic-pituitary-adrenal axis.
  • A 66-year-old woman exhibited symptoms such as weakness, weight loss, and poorly controlled hypertension, which led to the discovery of pheochromocytoma, a tumor that causes hormone overproduction, upon further testing.
  • After undergoing surgery to remove the tumor and receiving supportive treatment, her symptoms improved significantly, confirming the diagnosis of PCS rather than traditional Cushing's syndrome.
View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!