Endocrine Abstracts

Chronic exposure to hypercortisolism significantly impacts on patient’s health and health-related quality of life (HRQoL). We developed a disease-generated questio-nnaire to evaluate HRQoL in Cushing’s syndrome (CS) (CushingQoL); in 125 (104 females) patients recruited in Spain, France, Germany, The Netherlands and Italy, clinical and hormonal data were collected and correlated with results of the generic SF-36 questionnaire, a question on self-perceived general heal...

Introduction: LNSC has shown high sensitivity and specificity for the initial diagnosis of CD and detection of disease recurrence; however, the use of LNSC to monitor medical treatment of CD is not well established. The results of an exploratory analysis evaluating changes in LNSC in CD patients receiving long-acting pasireotide during a Phase III study (CSOM230G2304; Lacroix et al. Lancet Diabetes Endocrinol 2018) are reported here.Methods: Pat...

Acromegaly is a morbid condition mainly caused by overproduction of growth-hormone (GH) from a pituitary adenoma leading to excessive growth. Normalisation of insulin-like growth factor-1 (IGF1) is an important goal for the treatment of acromegaly. The second-generation somatostatin analog (SSA) long acting pasireotide (la-PAS) has recently been introduced for the management of patients uncontrolled by first-generation SSA. The ACRONIS study (CSOM230CIC05) will provide real-wo...

Introduction: Potent 11β-hydroxylase inhibitor osilodrostat provides cortisol level control in patients with Cushing’s syndrome (CS), as demonstrated by the LINC clinical development programme in Cushing’s disease (CD) patients.1 We report data from year 1 of the prospective observational LINC6 study (NCT05382156), evaluating long-term safety and efficacy of osilodrostat in CS patients during 3 years of routine clinical practice.<p class="abstext"...

Context: Somatostatin receptor ligands (SRLs) are the cornerstone medical treatment of acromegaly, however many patients remain uncontrolled under SRLs. Well-established predictive markers of response are needed.Objective: We aimed to explore the relationship between responsiveness to SRLs, on one hand, and somatostatin (SST)2A and 5 receptor expression, adenoma granulation, and T2-weighted MRI signal intensity (T2WSI), on the other hand.<p class="ab...

Background: Hyperphagia leading to craniopharyngioma-related obesity (CRO) is a common and serious sequel of treatments of craniopharyngiomas. The few therapeutic approaches that have been tested until now for the control of eating behaviour and weight have poor efficacy. Glucagon-like peptide-1 (GLP-1) analogues might be an option. Methods: This multicentre, randomised, double-blind superiority trial was conducted in France. Adults with CRO (BMI > 3...

Introduction: PBMAH is an heterogeneous disease from the clinical, hormonal, and morphological point of view. ARMC5 inactivating mutations have been reported as a cause of PBMAH. PDE11A4 variants have been associated with PBMAH and NR3C1 variants with bilateral adrenal incidentalomas.Aim: To analyse the frequency of ARMC5 pathogenic mutations and PDE11A4 and NR3C1 variants in PBMAH patients.<p cla...

Introduction: The Carney Complex is a multiple endocrine and non endocrine neoplasia mostly due to PRKAR1A mutations. Spectrum of manifestations and genotype-phenotype correlations have been previously described by retrospective studies. A prospective study evaluating the occurrence of the different manifestations was needed to precise the optimum follow-up.Methods: Multi-center national prospective study (Clinical Trials NCT00668291) including 70 patien...

Illicit expression of membrane receptors for circulating regulatory factors, such as gastric inhibitory polypeptide (GIP), luteinizing hormone (LH) and serotonin (5-HT) receptors, has been well documented in ACTH-independent macronodular adrenal hyperplasias (AIMAHs) causing Cushing’s syndrome. In addition, we have observed an abnormal expression of ACTH in some steroidogenic cells in two AIMAH tissues. The aim of the present study was to investigate the role of local pro...

Context: Ectopic GHRH secretion is a rare cause of acromegaly described mainly in isolated case reports.Setting: From the registry of the sole laboratory performing plasma GHRH assays in France, we identified cases of ectopic GHRH secretion presenting with acromegaly between 1983 and 2008.Patients: In total, 21 patients aged 14–77 years, from 12 French hospitals. Median GHRH was 548 ng/l (270–9779)....