hrp0094p1-185 | Pituitary B | ESPE2021

Puberty and pituitary-gonadal axis function after treatment for a childhood brain tumor

Rosimont Manon , Kariyawasam Dulanjalee , Samara-Boustani Dinane , Giani Elisa , Beltrand Jacques , Bolle Stephanie , Fresneau Brice , Puget Stephanie , Sainte-Rose Christian , Alapetite Claire , Pinto Graziella , Piketty Marie-Liesse , Brabant Severine , Abbou Samuel , Aerts Isabelle , Beccaria Kevin , Bourgeois Marie , Roujeau Thomas , Blauwblomme Thomas , Di Rocco Frederico , Thalassinos Caroline , Zerah Michel , Pauwels Christian , Rigaud Charlotte , James Syril , Busiah Kanetee , Simon Albanne , Bourdeaut Franck , Lemelle Lauriane , Guerrini-Rousseau Lea , Orbach Daniel , Touraine Philippe , Doz Francois , Dufour Christelle , Grill Jacques , Polak Michel , G. Gonzalez Briceno Laura ,

Introduction: Primary brain tumors are the second most common childhood malignancies, with an increasing survival rate over the years. Late effects on puberty and fertility alter survivors’ quality of life.Methods: We included 204 patients diagnosed with a primary brain tumor before 18 years, followed in pediatric endocrinology at the University Hospital “Necker-Enfants Malades” in Paris between January 20...

hrp0094p1-143 | Sex Endocrinology and Gonads B | ESPE2021

Current clinical practice of prenatal dexamethasone treatment in at risk pregnancies for classic 21 hydroxylase deficiency in Europe

Nowotny Hanna F. , Neumann Uta , Tardy-Guidollet Veronique , Ahmed S. Faisal , Baronio Federico , Battelino Tadej , Bertherat Jerome , Blankenstein Oliver , Bonomi Marco , Bouvattier Claire , de la Perriere Aude Brac , Brucker Sara , Cappa Marco , Chanson Philippe , Grinten Hedi L. Claahsen van der , Colao Annamaria , Cools Martine , Davies Justin H. , Gunther Dorr Helmut , Fenske Wiebke K. , Ghigo Ezio , Gravholt Claus H. , Huebner Angela , Husebye Eystein Sverre , Igbokwe Rebecca , Juul Anders , Kiefer Florian W. , Leger Juliane , Menassa Rita , Meyer Gesine , Neocleous Vassos , Phylactou Leonidas A , Rohayem Julia , Russo Gianni , Scaroni Carla , Touraine Philippe , Unger Nicole , Vojtkova Jarmila , Yeste Diego , Lajic Svetlana , Reisch Nicole ,

Background: Prenatal dexamethasone treatment (Pdex) has been used since the 1980s to prevent virilization in female offspring suspected to have congenital adrenal hyperplasia (CAH). However, due to lack of strong evidence for its best practice as well as limited data regarding long term adverse effects, use of dex is highly controversial. This study reveals the current medical practice regarding Pdex in female fetuses at risk of CAH due to 21 hydroxylase defic...

hrp0095p1-513 | Growth and Syndromes | ESPE2022

Long-term effectiveness and safety of childhood growth hormone treatment in Turner syndrome from two large observational studies

Backeljauw Philippe , Pietropoli Alberto , Rohrer Tilman

Patients with Turner syndrome (TS) have short stature, despite having normal growth hormone (GH) secretion. Treatment with recombinant human GH is recommended. The effectiveness and safety of Norditropin® (somatropin, Novo Nordisk) over ≤10 years of follow-up were investigated in two non-interventional studies: NordiNet® IOS (NCT00960128) and the ANSWER Program (NCT01009905). Of 2,409 children with TS, 2,377 were included in the full analysis s...

hrp0097fc5.6 | Diabetes and insulin 1 | ESPE2023

Post-Hypoglycemic Hyperglycemia Are Highly Relevant Markers For Stratification Of Glycemic Variability and Remission Status Of Pediatric Patients With New-Onset Type 1 Diabetes.

Harvengt Antoine , Pollé Olivier , Lysy Philippe

Aims: Recently, our team introduced the concept of post-hypoglycemic hyperglycemia (PHH) in the context that recovery of hypoglycemia impacts cardiovascular risk. GLUREDIA study aimed to evaluate whether PHH parameters correlated with glycemic homeostasis during the first year after type 1 diabetes onset and helped to distinguish pediatric patients undergoing partial remission or not.Methods: In the GLUREDIA study, longi...

hrp0097s6.1 | Neonatal endocrinology | ESPE2023

Unravelling the role of epigenetics in reproductive adaptations to early-life environment

Melamed Philippa

Adult reproductive function is affected by early life environments and experiences, pointing to a possible role for epigenetic modifications in directing an adaptive response. Using mouse and cellular models, we have been studying the mechanisms underlying the shorter reproductive lifespan experienced by Bangladeshi women who grew up in Bangladesh as opposed to the UK, a phenotype associated specifically with the greater immune challenges in Bangladeshi childhood and altered D...

hrp0092ha2 | Partial CRISPR/Cas9 IL1R1 & IFNGR1 Knock-Down Improves ß-Cell Survival and Function Under Cytokine-Induced Inflammation | ESPE2019

Partial CRISPR/Cas9 IL1R1 & IFNGR1 Knock-Down Improves β-cell Survival And Function Under Cytokine-Induced Inflammation

Daems Caroline , Vanderroost Juliette , Sokal Etienne , Lysy Philippe

Type 1 diabetes (T1D) is a chronic disease characterized by the autoimmune destruction of pancreatic β cells. This destruction is mediated by lymphocytes T helper and cytotoxic, and by the action of the pro-inflammatory cytokines IL1β, IFNγ and TNFa inside the islets of Langerhans. We propose a new approach to alleviate islet inflammation by targeting pro-inflammatory cytokine receptors. Our hypothesis is that the downregulation of inflammatory pathways ma...

hrp0092p3-84 | Diabetes and Insulin | ESPE2019

A Low-Carbohydrate Diet Improves Metabolic Control in a Type 1 Diabetic Child Without Side Effects

Klee Philippe , Stasinaki Aikaterini , Gozzi Tiziana , Schwitzgebel Valérie

Background and Aims: Despite intensive insulin treatment of type 1 diabetes (T1DM), metabolic control remains suboptimal, especially in children. In an attempt to optimize postprandial glycaemia, some families decrease the amount of carbohydrates contained in a meal. While "low-carbohydrate diets" may improve metabolic control in some selected populations, controversies remain around the risk of hypoglycemia and ketoacidosis and the impact of such diet...

hrp0089rfc3.2 | Diabetes and Insulin 1 | ESPE2018

Personalized and Predictive Medicine for Pediatric Diabetes Through a Genetic Test Using Next Generation Sequencing

Schwitzgebel Valerie , Klee Philippe , Dirlewanger Mirjam , Blouin Jean-Louis

Introduction: Monogenic diabetes (MD) accounts for at least 3% of all pediatric diabetes cases. MD is often misdiagnosed as type 1 or type 2 diabetes, because of its wide phenotypic spectrum. While clinical and biochemical parameters can suggest MD, a definitive diagnosis requires genetic analysis. We conducted a broad study to diagnose MD cases. Then, with the gained knowledge, we designed a new diagnostic to...

hrp0089p1-p162 | Growth & Syndromes P1 | ESPE2018

Comparing the Cumulative Dose of Growth Hormone Therapy Using Body Weight-Based Dosing Versus Body Surface Area-Based Dosing in Children with Turner Syndrome – Data from the ANSWER Study

Backeljauw Philippe , Geffner Mitchell , Ross Judith , Holot Natalia , Ostrow Vlady

Background and Objective: The American Norditropin Studies: Web-Enabled Research (ANSWER) Program is a long-term, US-based, non-interventional study designed to collect information on the effectiveness and safety of Norditropin® growth hormone (GH). From June 2002 to September 2016, 20,204 pediatric patients were enrolled by their treating physicians, including 1,003 patients with Turner syndrome (TS). This analysis compares cumulative GH doses when adjusting G...