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...

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: Improving physical manifestations of hypercortisolism is an important treatment goal for patients with Cushing’s disease (CD). In the Phase III LINC 3 study (NCT02180217), osilodrostat therapy, a potent oral 11β-hydroxylase inhibitor, rapidly normalised mean urinary free cortisol (mUFC) in most patients with CD and sustained control of mUFC over a median treatment period of 130 weeks (W). Here we describe concomitant improvements in physical manifestation...

Introduction: Osilodrostat decreases cortisol production by inhibiting 11β-hydroxylase, increasing adrenal hormones above the blockade. Here, we describe these effects of osilodrostat and associated adverse events (AEs). The efficacy and safety of osilodrostat in patients with Cushing’s disease (CD) were confirmed in the published Phase III, prospective LINC 3 study (NCT02180217).Methods: 137 patients with CD (mUFC >1.5x upper limit of norm...