Endocrine Abstracts

Purpose: Osteopathy is an emerging complication of acromegaly characterized by increased bone turnover, deterioration in bone microarchitecture and high risk of vertebral fractures (VFs). In somatostatin analog (SSA)-resistant patients (pts), Pegvisomant (PegV) and Pasireotide LAR (Pasi) are used for acromegaly treatment, but their effect on skeletal health is still not defined.Methods: In a longitudinal international multicenter study, we evaluated inci...

A personalized patient treatment regimen for endogenous Cushing’s syndrome (CS) should be developed by a specialized multidisciplinary team, taking patient values and preferences into consideration. Comorbidities, which may compromise patient health and QoL need to be addressed, in many cases concomitant with or even before CS-specific treatments to restore eucortisolemia. Treatment of endogenous CS is initially primarily surgical and aims at complete resection of the und...

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...

BackgroundData on clinical characteristics and natural history of patients with non-functioning pituitary microadenomas (NFPmA) is limited to small-scale studies.MethodsA retrospective evaluation of initial clinical presentation and natural history of patients with NFPmA (conservatively managed, years; 2004–2020) was undertaken. Initial symptoms, tumor size, and pituitary function were assessed. Exclusio...

11β-hydroxysteroid dehydrogenase type 1 (HSD-1) controls the intracellular cortisol pool that has access to cytosolic glucocorticoid and mineralocorticoid receptors. HSD-1 activity is elevated in patients with Cushing’s syndrome (CS).1 Patients with CS and constitutionally low HSD-1 activity showed no hypercortisolism-related symptoms despite very high 24-hour urine free cortisol.2, 3 A recent pilot trial of a HSD-1 inhibitor in patients with classical or mild CS sho...

Introduction: Although biochemical markers of acromegaly disease activity, including GH and IGF1, may fluctuate from day-to-day, biochemical treatment response in clinical trials is generally monitored using single-point analyses. Accordingly, longitudinal evaluations may assess patient status more accurately. In a phase 3 trial, oral octreotide capsules (OOC) demonstrated sustained composite endpoint GH and IGF1 response for ≤13 months in 151 patients with acromegaly pr...

Introduction: Osilodrostat is a potent, oral inhibitor of 11β-hydroxylase, the enzyme that catalyses final step of cortisol biosynthesis. In a phase II study, 15/19 patients treated with osilodrostat met the primary endpoint (normal urinary free cortisol (UFC) at 22 weeks); osilodrostat was generally well tolerated. This phase III study aims to confirm the efficacy and long-term safety of osilodrostat.Patients and methods: Adults (18–75 years) ...

Background: Dopamine and somatostatin receptors on corticotroph pituitary adenomas are targets for medical treatment of Cushing’s disease (CD). Data indicate synergistic effects between pasireotide and cabergoline in improving biochemical control rates and clinical features in patients with CD.Objective: To evaluate safety and efficacy of pasireotide alone or with cabergoline in patients with CD.Methods: Patients: adults with ...

The COVID-19 pandemic is significantly affecting the care of chronic conditions globally. Acromegaly is a rare chronic condition with a prevalence ranging between 2.8–13.7 cases/100.000 people. Acromegaly management requires a multidisciplinary team of health care professionals (HCPs), mainly including endocrinologists, neurosurgeons, and specialized endocrine nurses. Frequent consultations, particularly during early stages of the disease, and pre/post-surgery are necess...

Background: Medical treatment for acromegaly (characterized by excess growth hormone [GH] production) includes somatostatin receptor ligands (SRLs), dopamine agonists (DAs), and GH receptor antagonists (GHRAs). However, recent real-world United States (US) treatment evaluations are few. We present treatment patterns for patients receiving medications for acromegaly (1/1/2010–31/7/2022).Methods: De-identified data were extracted from MarketScan&...