Endocrine Abstracts

Pasireotide (Pas) is a multireceptor-targeted somatostatin analogue approved for the treatment of patients with Cushing’s Disease (CD) who fail or are poor candidates to surgery. Pas markedly improves signs and symptoms of the disease, reduces urinary free cortisol (UFC) up to its normalization in 55% of patients and pituitary tumour size in up to 100%. Here we present a patient with severe recurrent CD treated with Pas and showing opposite results between hormonal levels...

Patients: Nineteen acromegalic patients (8 females, 21-69 years-old, with macroadenoma, microadenoma or no evidence of pituitary tumor in 15, 2, 2, respectively) resistant to first generation somatostatin analogs (FG-SA) at high doses and/or intolerant to pegvisomant were switched to pasireotide LAR (PasLAR). Eleven had persistent disease after neurosurgery and two had also undergone radiosurgery (12 and 24 months before starting PasLAR). Six complained of acromegalic headache...

Background: Pasireotide is a multi-ligand somatostatin analogue licensed in a long-acting release formulation (PAS-LAR) for the treatment of acromegaly. The real-life reports with PAS-LAR are still scanty.Objectives: To assess the efficacy and safety of PAS-LAR in patients with acromegaly.Patients and methods: Prospective observational multi-centre study enrolling acromegalics evaluated before (baseline) and 1, 6, 12, 24, and 36 mo...

Introduction: Paragangliomas are rare neuroendocrine tumors, that manifest as painless, slow-growing masses, becoming evident only when symptoms of catecholamine overproduction or mass effects emerge. Paragangliomas exhibit a broad spectrum of characteristics, existing as solitary or multiple entities, and may be sporadic or hereditary. These tumors can be either benign or malignant and have origin in the sympathetic or parasympathetic tissues. Paragangliomas are commonly foun...

COVID-19 pandemic obliged physicians to find out alternative options to classical pathways, to lower viral spread and related dangers as well as to cope with redeployment of personnel and resources. We describe here two cases in whom surgery for adrenal Cushing’s syndrome (CS) and pheochromocytoma (PHEO) was deferred due to the unavailability of surgical facilities.Case no 1: A 69-yo woman was evaluated for CS. Type 2 diabetes mellitus was diagnosed...

Paragangliomas (PGLs) are rare neuroendocrine tumors. About 30–40% of these tumors are mutated in different susceptibility genes, including those encoding the different subunits of the succinate dehydrogenase, a complex involved both in the tricarboxylic acid cycle and in the oxygen transport chain. The aim of this project was to investigate whether SDHB mutations may account for alterations in cell metabolism and functions. Since PGL cell lines are not available, we used...

Metyrapone treatment in endogenous Cushing’s syndrome. Results from a prospective multicenter, open-label, phase III/IV study: PROMPTBackground: Metyrapone is a steroidogenesis inhibitor approved in Europe for the treatment of endogenous Cushing’s syndrome (CS) based on observational retrospective studies published over more than 50 years. We present data from the first prospective study designed to confirm metyrapone efficacy and good tolera...

Purpose: To genotype patients with head–neck paragangliomas (HNPGL) and evaluate the percentage and types of germ-line mutations in patients classified according to family history (FH) and clinical presentation.Design: In total, 101 consecutive patients with HNPGL were examined for mutations in SDHB, SDHC, SDHD. SDHAF2 and VHL genes by PCR/sequencing.According to a careful FH, clinical, laboratory (including meta...

BackgroundRetrospective studies led to European approval of the steroidogenesis inhibitor Metyrapone for the treatment of endogenous Cushing’s syndrome (CS). We prospectively showed good efficacy and safety of Metyrapone after 12 weeks (Wk12) treatment in the phase III/IV PROMPT study and now report results of an extension study (EXT) sponsored by HRA Pharma Rare Diseases.DesignThis was a single arm, ope...