hrp0094p2-98 | Bone, growth plate and mineral metabolism | ESPE2021

BUR-CL207: An Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Pediatric Patients from Birth to Less than 1 Year of Age with XLH.

Padidela Raja , Cheung Moira , Allgrove Jeremy , Bacchetta Justine , Semler Oliver , Heubner Angela , Schnabel Dirk , Emma Franceso , Nilsson Ola , Hogler Wolfgang , De La Cerda Ojeda Francisco , Quattrocchi Emilia , Linglart Agnes ,

Background: X-linked hypophosphatemia (XLH) is caused by mutations in PHEX which increases serum Fibroblast Growth Factor 23 (FGF23) concentrations leading to phosphate wasting and osteomalacia. Burosumab is a recombinant fully human IgG1 monoclonal antibody which selectively inhibits the activity of FGF23. In clinical trials burosumab demonstrated significant clinical improvements in radiological rickets severity, growth, and biochemistry among XLH c...

hrp0095p1-416 | Adrenals and HPA Axis | ESPE2022

The assessment of the usefulness of morning cortisol and DHEAS concentration levels in the diagnosis of central adrenal insufficiency in pediatric patients - preliminary results

Szczudlik Ewa , Wójcik Małgorzata , Stępniewska Anna , Januś Dominika , B. Starzyk Jerzy

The diagnosis of central adrenal insufficiency (CAI) is challenging. The most popular diagnostic tool in pediatric population remains low-dose (1 μg) cosyntropin (LDC) test. Nevertheless, there is still a need for the CAI markers that might be used on an outpatient setting as a first-line screening. In adult patients morning cortisol and dehydroepiandrosterone sulphate (DHEAS) has been postulated as a possible markers. The aim of the study was to evaluate the usefulness of ...

hrp0095p1-433 | Bone, Growth Plate and Mineral Metabolism | ESPE2022

Challenges in the management of infantile hypophosphatasia: revisiting the importance of supportive therapy in limited-resource settings

Fadiana Ghaisani , Tridjaja Bambang , B Pulungan Aman , RL Batubara Jose , Soesanti Frida

Background: Infantile hypophosphatasia (HPP) is an ultra-rare condition that may lead to debilitating morbidity and mortality. The prevalence of HPP in Indonesia is unknown. Enzyme replacement therapy or asfotase alfa, a bone-targeted recombinant alkaline phosphatase, can improve clinical outcome and prognosis of the patients, particularly those with severe type of HPP. The complications of HPP may be life-threatening and contribute to higher mortality in youn...

hrp0095p1-309 | Growth and Syndromes | ESPE2022

International Standard Growth Charts Overestimates Stunting Prevalence in Indonesia Compared to the Indonesian National Growth Chart

Natalia Hasibuan Susi , M Djer Mulyadi , A Andarie Attika , B Pulungan Aman

Background: Growth problems, such as stunting, are commonly found in daily pediatric practice. Stunting is a significant problem in developing countries, and is part of the Sustainable Development Goals (SDG) Goal 2 of ending hunger. In Indonesia, the rate of stunting in children under 5 years old is still considered high, despite improvement in quality of life due to rapid economic development. The rate of this improvement is unequal throughout the nation, wh...

hrp0092rfc2.3 | Bone, Growth Plate and Mineral Metabolism Session 1 | ESPE2019

Growth Hormone Effects on Metacarpal Bone Geometry and Bone Age in Growth Hormone-Deficient Children

Martin David D , Ranke Michael B , Henrik Thodberg Hans , Binder Gerhard

Purpose: To track the effects of growth hormone on bone geometry and maturation in children with isolated growth hormone deficiency (GHD).Methods: 299 left hand X-rays from 77 short prepubertal children (54 boys and 23 girls) with isolated GHD were analysed for changes in metacarpal thickness, width, length, medullary diameter, the Bone Health Index (BHI) and bone age (BA) from one year preceding until four years after s...

hrp0092p1-370 | GH and IGFs (2) | ESPE2019

Challenges Experienced in Delivering Growth Hormone Therapy in Children's with Prader Willi Syndrome in Birmingham Children's Hospital.

Kollurage D Udeni Anuruddhika , Barrett Tim , Jayamanne B D W , Krone Ruth

Prader Willi Syndrome (PWS) is a rare neuro-genetic disorder inherited as a result of lack of expression in 15q11-13 gene and 70% are paternally inherited. Characteristic features are dysmorphism, behavioural problems, infantile hypotonia, short stature, hypothalamic dysfunction, hyperphagia and morbid obesity. The long-term morbidity and mortality depend on hypothalamic dysfunction and obesity. While multidisciplinary care is essential, growth hormone (GH) is a recognized...

hrp0092lb-24 | Late Breaking Posters | ESPE2019

Abdominal Adiposity and Total Body Fat as Predictors of Cardiometabolic Health in Pre-Pubertal and Pubertal Youth

Jin Binghan , Derraik José G B , Fu JunFen , Lin Hu , Yuan Jinna , Dong Guanping

Objective: We aimed to investigate the usefulness of abdominal adiposity and total body fat as predictors of cardiometabolic health, especially insulin sensitivity, in children and adolescents.Methods: Participants were 479 children and adolescents with obesity (322 boys and 157 girls) aged 3 to 18 years attending the Children's Hospital at Zhejiang University School of Medicine (Hangzhou, China). Participants underw...

hrp0089fc15.5 | Growth and Syndromes | ESPE2018

Utility of BDNF and MMP-1 as Markers of Cardiometabolic Risk in Turner Syndrome Girls

Błaszczyk Ewa , Miłosz Lorek , Francuz Tomasz , Gieburowska Joanna , Gawlik Aneta

Background: It remains unclear whether cardiometabolic and vascular risks in Turner syndrome (TS) are the consequence of unidentified intrinsic factors or, conversely, the result of modifiable risk factors, such as overweight. New markers that could explain the pathogenesis of metabolic complications are under investigation.Objective: The comparison of the selected biochemical cardiometabolic risk markers between TS patients and healthy controls.<p c...

hrp0089p2-p124 | Fat, Metabolism and Obesity P2 | ESPE2018

Relationships of Dietary Intake and Sugar Rich Products Consumption with Hepatic Fat Content and Insulin Resistance among Children with Overweight/Obesity: The PREDIKID Study

Aranaza Lide , Diez-Lopez Ignacio , Medrano Maria , Oses Maddi , Huybrechts Inge , Ortega Fran B , Labayen Idoia

Pediatric non-alcoholic fatty liver disease (NAFLD) has increased in parallel with childhood obesity. Dietary habits, particularly products rich in sugars, may influence both hepatic fat content and insulin resistance. Hence, the aim of the current study was to examine the associations of the consumption of dietary foods (cereals, fruits and vegetables, meat and meat products, dairy products, fish and shellfish, total and added sugars) and composition (macronutrients and fiber...

hrp0089p2-p219 | GH &amp; IGFs P2 | ESPE2018

Baseline Demographics of the TransCon GH Phase 3 heiGHt Trial

Beckert Michael , Karpf David B , Shu Aimee , Lin Zhengning , Leff Jonathan A

Background: TransCon GH is a novel sustained-release recombinant human GH (somatropin) prodrug in development for children with GH deficiency (GHD). It is designed to release unmodified GH and intended to provide comparable efficacy, safety, tolerability, and immunogenicity to daily GH with once-weekly dosing. Based on results from a phase 2 trial, which demonstrated comparable efficacy (annualized height velocity for TransCon 0.21 mg GH/kg per week of 12.9 cm/year vs 11.6 cm/...