hrp0089p2-p260 | Growth & Syndromes P2 | ESPE2018

An Irish Regional Study of Paediatric Growth Hormone Deficiency (CO-GHD): Classification of Causes and Factors Associated with Persistent GHD at Transition

Grace Mariana , Joyce Caroline , Morissey Rose , Moore Michael , O'Connell Susan

Childhood-onset growth hormone deficiency (CO-GHD) is topical at present due to the increasing understanding of underlying genetic aetiologies, influence on childhood growth, and future effects on adolescence and adult health. There is no previous Irish data of this group of children.Methods: A retrospective cohort study over 2 years (2013-2015) including all children diagnosed with GHD who received recombinant growth hormone treatment (rGH). Predictors ...

hrp0086fc2.4 | Bone & Mineral Metabolism | ESPE2016

In vitro Evidence that Growth Plate Chondrocytes Differentiate into Perichondrial Cells

Spath Stephan-Stanislaw , Andrade Anenisia , Chau Michael , Nilsson Ola

Background: During early bone formation, mesenchymal stem-cells condense and differentiate into collagen type 2-expressing chondrocytes which form the cartilaginous bone anlagen. This anlage becomes enclosed by the perichondrium. The perichondrium consists of two layers, the inner cambium layer containing chondrocyte- and osteo-precursor cells and the outer fibrous layer important for mechanical and structural support. While the regulation of the growth plate is increasingly w...

hrp0086rfc8.6 | Growth: Clinical | ESPE2016

A Six-Month Safety and Efficacy Study of TransCon hGH Compared to Daily hGH in Pre-Pubertal Children with Growth Hormone Deficiency (GHD)

Chatelain Pierre , Malievsky Oleg , Radziuk Klaudziya , Senatorova Ganna , Beckert Michael

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...

hrp0086p1-p603 | Growth P1 | ESPE2016

Pediatric Phase 2 Data Demonstrate that TransCon hGH Has an Anti-hGH Immunogenic Profile that is Comparable to Daily hGH

Gilfoyle David , Pihl Susanne , Chatelain Pierre , Beckert Michael

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...

hrp0086p1-p823 | Syndromes: Mechanisms and Management P1 | ESPE2016

Effects of a Stressful Environment (SE) on Height, BMI and Menarche

German Alina , Mesch Gustavo , Shmoish Michael , Hochberg Ze'ev

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...

hrp0084p2-407 | GH & IGF | ESPE2015

A Phase 2, 6-Month, Randomised, Active-Controlled, Safety and Efficacy Study of TransCon hGH Compared to Daily Human GH in Children with GH Deficiency

Chatelain Pierre , Malievsky Oleg , Radziuk Klaudziya , Senatorova Ganna , Beckert Michael

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...

hrp0084p3-1083 | Hypo | ESPE2015

Cholestatic Hepatopathy and Hypoglycaemic Seizures as Primary Manifestation of Hypocortisolism in Infancy

Saupp Peter , Fried Michael , Bergmann Carsten , Meissner Thomas , Kummer Sebastian

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 ...

hrp0098p1-313 | Late Breaking 2 | ESPE2024

Do We Need Seasonal Thyroid Hormone Reference Values in Pediatric Endocrinology? Results from a Population-Based Study

Hirtz Raphael , Thamm Michael , Kuhnert Ronny , Thamm Roma

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...

hrp0086fc13.2 | Management of Obesity | ESPE2016

Measurement of Immunofunctional Leptin to Detect Patients with Functional Leptin Deficiency

Wabitsch Martin , Pridzun Lutz , Ranke Michael , Flehmig Bertram , Fischer-Posovszky Pamela , Moss Anja , Schaab Michael , von Schnurbein Julia , Kratzsch Jurgen

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...

hrp0082p3-d1-982 | Thyroid | ESPE2014

Preliminary Result and Normative TSH Values for Healthy Nigerian Newborn Children

Yarhere Iroro , Oduwole Abiola , Suwaid Salma , Yahaya Alkali , Ibekwe Maryanne , Adeniran Kayode , Fetuga Bolanle , Elusiyan Jerome , Idris Hafsat W , Jaja Tamunopriye , Adesiyun Omotayo , Jarrett O O

Background: Congenital hypothyroidism (CH) is the commonest congenital endocrine disorder in the world and also the commonest most preventable cause of mental retardation. Screening is mandatory in developed countries, but none exists in sub-Saharan country. We present a preliminary report of the first Nigerian screening for CH.Objective and hypotheses: To screen normal newborn babies in different regions in Nigeria and to determine the normal range of T...