Endocrine Abstracts

The management of adults with congenital adrenal hyperplasia due to classic 21-hydroxylase deficiency (21OHD) is a challenging balance of hormone replacement, disease control, and avoidance of adverse effects. The approach should emphasize the clinical evaluation and patient goals, and laboratory tests are used secondarily as ancillary data. Upon transition from pediatric to adult care, the concerns shift from growth and pubertal development to fertility, neoplasia formation, ...

In the 60 years since primary aldosteronism (PA) was described, our understanding of its pathophysiology and approaches to diagnosis and treatment has improved remarkably. Despite this progress, <1% of patients with PA are ever screened for this condition, which accounts for 5–8% of hypertension (HTN). The resistance to screening for PA probably derives primarily from the complexities and uncertainties inherent in its evaluation and management. How many types of PA ex...

Dr Richard J Auchus is Professor of Pharmacology and Internal Medicine in the Division of Metabolism, Endocrinology, and Diabetes at the University of Michigan and Director of the Diabetes, Endocrinology, & Metabolism Fellowship Program at Michigan. He did postdoctoral work and training at the University of California, San Francisco prior to joining the faculty at UT Southwestern in Dallas. He served as Acting Chi...

Background: Osilodrostat is an oral inhibitor of 11β-hydroxylase (CYP11B1), the enzyme that catalyses the final step in cortisol biosynthesis. In a 22-week, phase II study, osilodrostat treatment normalised mean urinary free cortisol (mUFC) in 78.9% (15/19) of patients with uncontrolled Cushing’s disease and was well tolerated. The present phase 3 study is designed to confirm the safety and efficacy of osilodrostat in patients with uncontrolled Cushing’s disease...

Background: Cushing’s syndrome (CS) has numerous comorbidities, including diabetes mellitus (DM). Levoketoconazole is a ketoconazole stereoisomer in clinical trials for treatment of CS.Methods: SONICS is a prospective, open-label, phase 3 maintenance-of-benefit study in adults with confirmed CS and mean urinary free cortisol (mUFC) of ≥1.5x upper limit of normal (ULN). Repeated hospitalization due to hyperglycemia or any complication related t...

Background: The phase 3, open-label SONICS study of levoketoconazole demonstrated sustained reduction in mean urinary free cortisol (mUFC) in adults with endogenous Cushing’s syndrome (CS). The extended evaluation phase (Ext) of SONICS reported here further assessed long-term safety, tolerability, and benefit/risk of this treatment.Methods: SONICS consisted of dose-titration (150 – 600 mg BID to attain maximally tolerated dose for mUFC normal...