ESPE Abstracts

Background: Multiple daily injections of insulin for children and young people with diabetes can be very daunting and, some have a phobia of needles. As a result, for some individuals, the use of an i-port device can be life-changing and help with their diabetes management. The i-port is an injection port that enables diabetes patients to administer their insulin without having to puncture their skin multiple times in a day. In most cases, the i-port can stay ...

Background: Diabetic ketoacidosis (DKA) in children and young adults carries significant morbidity and mortality relating to complications such as cerebral oedema. Acute kidney injury (AKI) is a rare but potentially fatal complication of DKA. We present three cases of DKA complicated by AKI.Case 1: A 9-year-old girl presented with severe DKA at diagnosis. She was treated with intravenous fluids and insulin as per protocol. She had oliguria and haematuria...

Introduction: The role of genetics in obesity is a much under discussed area. Whilst it is undeniable that environmental factors play a major role in obesity in most cases, there is a small proportion of cases where genetic mutations are the main underlying cause. This includes novel monogenic conditions involving mutations in the Melanocortin-4 receptor (MC4R) signalling pathway. In a healthy individual, the post-meal increase in leptin (LEP) stimulates melan...

Introduction: Insulin stress test (IST) to diagnose growth hormone (GH) deficiency is rarely used by paediatric endocrine teams in the United Kingdom, due to concerns over safety. We share our experience of undertaking modified IST in children, with regards to safety and outcomes.Methods: We adapted the protocol for insulin stress test, proposed by Galloway et al to undertake pituitary stimulation test for our c...

Background: Long-term treatment with recombinant human growth hormone (r-hGH) is often challenging for children and adolescents, their caregivers and healthcare professionals (HCPs), as treatment requires daily injections over several years. Connected digital health devices facilitate this by automating the injection process to improve comfort, reduce anxiety and collect GH treatment data so that accurate adherence information is available to HCPs in real-time...

Background: Silver–Russell syndrome (SRS; OMIM 180860) is a genetically and clinically heterogeneous low birthweight syndrome characterised by poor postnatal growth and a number of variable dysmorphic features. Small-for-gestational age infants in general have an increased risk of metabolic complications, some initially occurring in late childhood and adolescence.Objective and hypotheses: To identify i) response to GH based on genotype and ii) devel...

Background: Prader-Willi syndrome (PWS) is a rare genetic neurobehavioral-metabolic disorder characterized by hyperphagia and behavioral/psychological complications. With no approved therapies to treat hyperphagia, disease management requires strict dietary and environmental controls to restrict access to food. DCCR is an oral, once-daily medication currently under development for the treatment of PWS.Objective: The obje...

Background: Prader-Willi syndrome (PWS) is a rare genetic neurobehavioral-metabolic disorder characterized by hyperphagia and behavioral/psychological complications. No approved therapies exist for treating hyperphagia in patients with PWS. DCCR is an oral, once-daily medication currently under development for the treatment of PWS.Objective: The objective was to determine the efficacy of investigational DCCR on hyperphag...