ESPE Abstracts

Background: TransCon hGH is a long-acting prodrug of recombinant human growth hormone (hGH) that releases fully active unmodified hGH into the blood compartment. This presentation will detail the final safety and efficacy results of TransCon hGH in a Phase 2 study in children with GHD over a treatment period of six months.Objective and hypotheses: The objective of this Phase 2 study in GHD was to investigate 1) safety and tolerability, 2) pharmacokinetic...

Background: TransCon hGH, is a once-weekly prodrug releasing unmodified hGH, for the treatment of Growth Hormone Deficiency (GHD) in children and adults. To date, TransCon hGH has demonstrated comparable efficacy, safety and anti-hGH immunogenic profile to daily hGH, with no occurrence of neutralizing antibodies.Objective and hypotheses: Protein-based therapies may be associated with generation of drug-specific antibodies, which may impact efficacy, part...

Background: Children’s exposure to stress predicts poor health. Poor growth and maturation are recognized indicators of poor health.Objective and hypotheses: SE correlates with height, BMI and menarche. We correlated seven indicators of SE with countries’ average adult height, BMI and menarche age.Method: Data for 57 countries of average men and women’s height, BMI and menarche age were collected from WHO report. The...

Background: There is currently limited data published on the prevalence of diabetic eye disease in children and young people (CYP) with type 1 diabetes mellitus (T1DM), yet diabetic retinopathy remains one of the commonest causes of blindness in young adults.Objectives: To determine the risk and prevalence of diabetic retinopathy and to evaluate the risk factors associated with diabetic retinopathy in CYP with T1DM.Methods: All CYP...

Background: TransCon human GH (hGH) is a long-acting prodrug of recombinant hGH that releases fully active unmodified hGH. TransCon hGH was shown in phase 1 studies in healthy volunteers and a phase 2 study in adults with hGH deficiency to: i) be safe and well tolerated, ii) provide dose-dependent hGH levels, with same peak level and exposure compared to daily GH, iii) be suitable for a once-weekly dosing regimen, and iv) induce an IGF1 response within the normal range. This o...

Background: Cholestatic hepatopathy is a rare but serious threat to neonates and young infants. Their immature hepatic excretory function predisposes for severe and rapidly progressive hepatic injury. Because of the wide range of possible etiologies, it is often difficult to make an accurate diagnosis. One rare endocrine cause can be primary or secondary hypocortisolism.Case presentation: i) A 7-week-old term-born female with cholestasis, elevated liver ...

Studies in adults provide convincing evidence of seasonal fluctuations in thyroid hormone levels. However, the evidence in children and adolescents is less consistent, potentially due to unaddressed confounding and the use of statistical methods not well-suited to investigate this specific issue. Using the baseline data from KiGGS (German Health Interview and Examination Survey for Children and Adolescents), an analysis was conducted among 6,071 participants aged 6 to 17 years...

Background: Recently, patients with severe obesity have been described due to functional leptin deficiency. This new entity is characterized by high immunoreactive levels of circulating leptin (Lep), but a reduced bioactivity of the hormone due to defective receptor binding (N Engl J Med 2015;372:48–54). Since these patients can be successfully treated with human recombinant leptin (metreleptin), a diagnostic tool to detect functional leptin deficiency is needed.<p cl...

Background: Rare metabolic bone diseases (RMBD) are at the crossways of Endocrinology, Nephrology, Orthopaedic surgery and Rheumatology. Most of RMBD lead to short stature, bone pain, tooth anomalies, leg deformities, bone fragility and disability of variable importance. The organization of care varies immensely amongst European countries. Several European networks dedicated to RMBD already exist, supported by societies such as ESPE, ECTS or research grants, albeit their focus...

Background: In 2017 the American Academy of Pediatrics has introduced a new guideline (AAP 2017) to diagnose arterial hypertension in children, as the blood pressure thresholds for adults had been lowered before. There is a controversy about these new reference levels as other societies have not followed these recommendations. We studied the impact of the new AAP 2017 guideline on prevalence of arterial hypertension (HTN) in children with Type 1 diabetes melli...