Endocrine Abstracts

Introduction: Subcutaneous (sc) pasireotide effectively reduces cortisol levels and is generally well tolerated in Cushing’s disease (CD) patients, as demonstrated by a Phase III study (NCT00434148). We report data from a non-interventional, multinational study (NCT02310269) evaluating long-term safety and efficacy of pasireotide sc for CD patients.Methods: Adults with CD, for whom surgery has failed or is not an option, were analysed by time of pas...

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...

Dopamine agonists (DAs), used as first line therapy in patients with macroprolactinomas, and antipsychotics have opposite effects on dopamine receptors (D2R). In patients with severe psychiatric conditions treated with antipsychotics, the rare occurrence of a macroprolactinoma, particularly with optic chiasm compression, represents a therapeutic challenge. Indeed, on one hand, antipsychotics by their antagonistic effect on D2R, could decrease or even abolish the effects of DAs...

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...

Introduction: Germline inactivating mutations of the protein kinase A (PKA) regulatory subunit RIα (the PRKAR1A gene) cause primary pigmented nodular adrenocortical disease (PPNAD); other cyclic AMP (cAMP) signalling defects have been associated with bilateral adrenocortical hyperplasia (BAH), cortisol-producing adenoma (CPA) and related lesions. Recently, PRKACA somatic mutations were detected in single, sporadic CPAs in approximately 40% of patients wi...

Introduction: The PAOLA study in patients with inadequately-controlled acromegaly (n=198) demonstrated superior efficacy of pasireotide long-acting release (LAR; 40 mg/60 mg) in biochemical control (GH <2.5 μg/l and normalized IGF-1) vs continued treatment with octreotide LAR 30 mg/lanreotide Autogel 120 mg (15.4% and 20.0% vs 0%). Here we report preliminary data from the extension phase of PAOLA at wk28.Methods: Pasireotide-LAR (40 mg/...

Background: Some patients with acromegaly do not achieve biochemical control despite receiving maximum-approved doses of currently available somatostatin analogues. This 24-week, randomized study assessed the multireceptor-targeted somatostatin analogue pasireotide LAR vs octreotide LAR/lanreotide Autogel in patients with inadequately controlled acromegaly.Methods: Eligible patients: ≥18 years with mean GH levels ≥2.5 μg/l and IGF1 level...

Background: Constitutional duplications of the PRKACA gene locus have been described as responsible for adrenal Cushing’s disease.The objective here was to evaluate the results of its systematic screening in bilateral adrenal nodular disease and to specify the associated phenotype.Methods: Between 2020 and 2023, 440 consecutive index cases with macronodular or micronodular adrenal hyperplasia or Carney Complex (CNC) w...

Background: Papillary craniopharyngiomas (PCPs) are driven by V600E BRAF mutations in 95% of cases. Recently, combined anti-BRAF/MEK targeted therapy (TT) has emerged as a potential treatment in aggressive PCPs. However, standardized data on large cohorts are still lacking. Our study aimed to assess the real-life efficacy and safety of TT in patients with PCPs.Methods: This was a retrospective national multicenter study involving patients with V600E BRAF...