Endocrine Abstracts

The primary aim of this study, the largest European, single evaluation, observational trial to date, was to evaluate the impact of acromegaly on health-related quality of life (HRQoL) in patients with biochemically documented active disease, receiving Sandostatin® LAR® at a dose prescribed by their physician for at least 3 months. Secondary objectives were to investigate the relationships between HRQoL and subpopulations based on exploratory var...

Non-functioning pituitary adenomas (NFPA) are the most common pituitary tumours. They usually come to medical attention because of a mass effect and/or hypopituitarism. Tumour shrinkage during therapy with either dopamine agonists (DA) or somatostatin analogues (SSA) has been previously reported in some cases; however, response of NFPA to medical treatment is still poor and unpredictable. Our aim was to explore the molecular mechanisms underlying this lack of efficacy through ...

Background: Management of patients with CAH remains challenging. There is increasing evidence to suggest that failure to optimize treatment during childhood not only affects final height but also leads to psychological and psychiatric problems, reflecting an underlying effect on neural development. Previous qualitative structural T2-weighted MRI studies have identified white matter hyper-intensities in up to 46% of CAH patients. The nature and functional relevance of these abn...

Background: Complications of excess weight (CEW) clinics were commissioned by NHSE in 2021 to be leaders in the field of paediatric obesity using innovative models to deliver the highest quality patient care. Technological approaches offer a widely accessible tool which could potentially complement current clinical models of care. This study aims to explore children and young peoples (CYP) views on whether digital technology could/should be used to enhance CEW...

Background: Fibroblast growth factor 23 (FGF23) is a key protein in bone homeostasis regulatingserumphosphateand calcitriol (1,25(OH)2D3) concentrations. Analytical performance and reference ranges of FGF23 assays differ by molecule assayed (intact or C-terminal FGF23) and manufacturer. We aim to establish reference interval values for both cFGF23 and iFGF23 in a paediatric population.Methods: Writt...

Background: Prior to COVID-19, HbA1C was performed in clinic as a point of care test. As a service development project driven by the changes to our service from COVID-19 we offered either a hospital drive through point of care HbA1C test or the validated VAMS collection method to obtain HbA1C at home.Objective: Research ethics approval was obtained to assess patient/carer acceptability of these collection methods.Methods: Drive thr...

Background: Children and young people (CYP) with Type 1 diabetes are offered 4 face to face appointments annually. During the COVID-19 pandemic these were delivered virtually.Objective: We conducted a survey to assess patient and carer satisfaction with the remote diabetes service. Methods: Research ethics approval was obtained. The survey was distributed to 280 CYP and their families; one response per family was collected. Data on...

For all ERCUSYN investigatorsBackground: Patients with Cushing’s syndrome (CS) have an increased risk of developing venous thromboembolic complications. There is currently no standard practise for thromboprophylaxis in CS patients.Aim: To study the details of VTE in patients included in The European Registry on Cushing’s syndrome (ERCUSYN) and study various thromboprophylaxis protocols used among the centres where VTE&#14...

Background: TMEM38B is a ubiquitously expressed monovalent cation-specific channel protein hypothesized to play a role in intracellular calcium homeostasis. To date, only two unique recessively inherited exon deletions in TMEM38B have been reported in 17 individuals with osteogenesis imperfecta (OI). Data on bone histomorphometry and bone material property have not previously been presented.Cases: Targeted next generation sequencing was performe...

ATL1103 is a second generation antisense 20mer intended to inhibit expression of the GH receptor (GHR) gene. Phosphorothioate and 2′-O-methoxyethyl modifications to nucleotides increase its plasma half-life and affinity for the target RNA to allow post-hybridization RNaseH degradation. We previously reported a phase 2, randomised, open-label, parallel group study of ATL1103 in 26 patients with acromegaly which demonstrated a fall in serum IGF-I of 26% with 200 mg twice w...