Endocrine Abstracts

Introduction: Hypercortisolaemia is characterised by an increased risk of cardiovascular disease (CVD), either through a direct action on the myocardium or by affecting traditional cardiovascular risk factors. The electrocardiogram (ECG) is the initial examination to assess the structural and functional characteristics of the myocardium.Aim of the study: To investigate whether the metabolic and cardiovascular features of Cushing’s disease (CD) are a...

Background: Cyclical Cushing’s syndrome has been considered to be a rare clinical entity, characterised by periodic increases in cortisol levels followed by regression of the Cushing’s syndrome. The cycles of hypercortisolism may occur before the establishment of the diagnosis, rendering actual diagnosis difficult, or may occur after inadequate or ineffective treatment and affect disease management. The aim of this study was to investigate the prevalence of cyclicity...

Inherited growth hormone insensitivity (GHI) is usually caused by mutations in the GH receptor (GHR). Patients present with short stature associated with a mid-facial hypoplasia (Laron facial features) or with normal facial appearance. We previously described an intronic mutation in the GHR gene (A−1 to G−1 substitution in intron 6) resulting in the activation of a pseudoexon (6Ψ) in four related GHI patients with normal facial fea...

Medullary thyroid carcinoma (MTC) is a rare malignancy which has often metastasised at time of diagnosis. Surgical resection represents the only prospect for cure. However, debulking neck surgery may be beneficial in advanced cases. Prophylactic surgery is increasingly undertaken in asymptomatic patients with known mutations in the RET oncogene. The aim was to describe the outcome following initial surgical treatment for MTC at our institution. We performed a retrospe...

Long-acting somatostatin receptor ligands (LA-SRLs) are a first line medical treatment for acromegaly but require monthly parenteral administration. Paltusotine (CRN00808) is a nonpeptide, small molecule somatostatin type 2 (SST2) receptor agonist with high oral bioavailability (70%), suitable for once daily, oral dosing. The recently reported results of the ACROBAT Edge study (NCT03789656) suggest that patients with acromegaly treated with injected SRLs can switch to once dai...

Objective: Head-to-head data comparing selpercatinib, a highly selective and potent RE arranged during Transfection (RET) kinase inhibitor with CNS activity, to standard treatment of RET-altered medullary thyroid cancer (MTC; cabozantinib or vandetanib) is currently unavailable. This post-hoc comparative effectiveness analysis of data from the phase 1/2 LIBRETTO-001 trial compared investigator-assessed outcomes among patients with RET</e...

Background: Paltusotine is an investigational oral, once-daily, small molecule somatostatin receptor type 2 agonist in development for the treatment of acromegaly and carcinoid syndrome associated with neuroendocrine tumors.Methods: This multicenter, open-label, phase 1 study enrolled participants with mild, moderate, and severe hepatic impairment based on Child-Pugh classification and matched healthy participants. A single 20-mg oral dose of paltusotine...

Background: Peptide Receptor Radionuclide Therapy (PRRT) is effective for gastroenteropancreatic (GEP) neuroendocrine tumors (NETs) and received FDA approval after demonstrating improved progression-free-survival (PFS) in advanced midgut NETs. We investigated a two-decade experience with PRRT, hypothesizing that PRRT confers PFS and overall-survival (OS) advantages in patients who progress after surgery.Methods: A single-institutional NET database was re...

Background: Neuroendocrine tumors (NETs; ~2% of all malignancies) commonly arise from the GI tract, pancreas, and lung and often present with metastatic disease. The PI3K/Akt/mTOR pathway is implicated in the pathogenesis and progression of NETs. The oral mTOR inhibitor (mTORi), everolimus, is approved for treatment of patients with NETs of the GI tract, lung, or pancreas. However, due to the rarity and heterogeneity, nonspecific clinical symptoms, and unique indolent biology,...

Introduction: Adrenal venous sampling (AVS) is the criterion standard method of lateralisation in primary aldosteronism (PA). Despite this pivotal role, there is limited consensus and significant variability between centres related to many of the technical aspects of AVS. In this study, we sought to address whether variations in two different technical aspects of AVS altered parameters and interpretation, namely: 1. Peripheral sample site 2. Method of adrenal vein (AV) venesec...