Endocrine Abstracts

Glioblastoma (GBM) is a highly aggressive form of brain cancer with a median survival time of 12–15 months from diagnosis. Standard therapies utilise a combination of radiotherapy, chemotherapy, and surgery. Patients also receive high doses of the potent anti-inflammatory glucocorticoid (Gc), Dexamethasone (Dex).Recent studies show that patients receiving the highest dose of Dex also have reduced survival time. Defining pathways under Gc control relevant to GBM is necessa...

The overproduction of ACTH, in conditions such as Congenital Adrenal Hyperplasia (CAH) leads to significant morbidity. Current treatment with glucocorticoids does not adequately suppress plasma ACTH, resulting in excess adrenal androgen production. At present, there is no effective medical treatment that would directly block ACTH action. Such a therapy, especially one that can be orally administered, would be of great clinical value allowing a ‘block and replace’ tre...

Introduction: TNDM due to genetic aberrations at 6q24 is the commonest cause of diabetes presenting within the first week of life. TNDM predisposes to diabetes mellitus in later life. We report on the clinical presentation of the largest worldwide cohort of 6q24 TNDM cases.Results: 164 cases were analysed. The mean age of presentation was 8 days with a mode of 1 day and maximum age of presentation of 90 days. On average, babies recovered by 4.5 months wi...

Objective: To characterize patients with GH deficiency associated with lymphocytic hypophysitis (LyH).Method: Patients with a diagnosis of LyH were identified in the KIMS database. The responsible clinicians were asked to confirm the diagnosis and to provide more detailed information on the patient by filling in a questionnaire.Results: One hundred and fifty patients with a diagnosis of LyH were identified in the database and 100 c...

Background: Neuroendocrine tumours (NETs) are heterogeneous cancers with varied survival, arising mainly from the gastroenteropancreatic tract. Delayed diagnosis and mislabelling are widely recognised internationally in midgut NETs with little data describing how to improve diagnosis times. In 2017, transformation of the South Wales NET Service included a change to a gastroenterology-led service model alongside education and forming working relationships with gastroenterologis...

Paltusotine is an investigational oral, once-daily, non-peptide, SST2 agonist in development for the treatment of acromegaly and neuroendocrine tumors. Interim analysis results from subjects with acromegaly treated with paltusotine for up to 2 years in ACROBAT Advance (NCT04261712), an ongoing, open-label extension study are reported here. Prior to Advance, subjects previously completed one of two Phase 2 parent studies, Evolve (NCT03792555, with normal IGF-1 using injected lo...

CRN04894 is a potent, orally bioavailable nonpeptide that is a highly selective antagonist for melanocortin type 2 receptor (MC2R). This receptor is found exclusively in the adrenal cortex and is the primary mediator of adrenal activation. We report results from a randomized, double-blinded, placebo-controlled (6 active:3 placebo/cohort), multiple ascending dose (40, 60, and 80 mg) study in health volunteers evaluating safety, pharmacokinetics, and pharmacodynamics of oral, on...

Introduction: LAN, a somatostatin analogue, is a common first-line treatment for NETs. LAN comes as a pre-filled, ready-to-use syringe, administered every 4 weeks. In the UK, patients may receive LAN at home. The aim of PREF-NET was to generate real-world evidence describing patient’s experience of LAN administered in homecare and hospital settings, and the associated impact of treatment setting on other areas of patients’ lives.Methods: PREF-N...

Pheochromocytomas and paragangliomas (PPGLs) are rare neuroendocrine tumours, which arise from neural crest (NC)-derived structures: the adrenal medulla and the paraganglia. Around one third of PPGLs are associated with inherited cancer susceptibility genes, the highest rate among all tumour types. Currently, the only diagnostic criterion for malignant disease is the presence of metastasis and no molecular or histological features have been identified that help predict risk. A...

Background: Transformation of the South Wales NET service in late 2017 introduced new specialised staff, gastroenterology-led clinics and MDTs with a particular emphasis on educating referring specialties on NETs. Data has shown patient experience, satisfaction rates, quality of life and gastrointestinal symptom scores have all improved. This study aims to describe findings from new NET service data for: referrals into the service, diagnosis times after symptoms, and initial s...