hrp0097rfc6.5 | Pituitary, neuroendocrinology and puberty 1 | ESPE2023

Efficacy and security of gonadotropin treatment in adolescents with congenital hypogonadotropic hypogonadism

Sayol-Torres Laura , González Judith , Gonzalez Núria , Campos Ariadna , Mogas Eduard , Yeste Diego , Clemente Maria

Objective: To describe efficacy and security of treatment protocol with gonadotropins in adolescents with hypogonadotropic hypogonadism (HH).Methods: Prospective study of patients with HH who received hGH and rhFSH in puberty. HH diagnosed during first months of life or in adolescence (testicular volume<4cc in >16 year-old with FSH<1.2UI/L, testosterone<40ng/dL and GnRH-test with LH-peak<6UI/L)<p class...

hrp0092rfc14.1 | Adrenals and HP Axis | ESPE2019

PAPP-A2 Deficiency Results in Sex-Dependent Modifications in Hypothalamic Regulation of Energy Homeostasis

Rivera Patricia , Vargas Antonio , Bonsón Javier , Christians Julian , Rodríguez de Fonseca Fernando , Chowen Julie , Suárez Juan , Argente Jesús

Pregnancy associated plasma protein (PAPP)-A2 is an insulin-like growth factor (IGF) binding protein (BP) protease that regulates IGF-1 availability affecting postnatal growth. Mutations in human PAPP-A2 cause short stature and changes in bone size and mineral density. The present study aimed to characterize the effects of constitutive Pappa2 gene deletion on hypothalamic regulation of energy homeostasis in adult male and female mice. In addition to being sho...

hrp0089fc2.1 | Bone, Growth Plate &amp; Mineral Metabolism 1 | ESPE2018

Burosumab, a Fully Human anti-FGF23 Monoclonal Antibody, for X-linked Hypophosphatemia (XLH): Sustained Improvement in two Phase 2 Trials in Affected Children 1–12 years old

Linglart Agnes , van't Hoff William , Whyte Michael P. , Imel Erik , Portale Anthony A. , Boot Annemieke , Hogler Wolfgang , Padidela Raja , Mao Meng , Skrinar Alison , Martin Javier San , Carpenter Thomas O.

In XLH, excess fibroblast growth factor 23 (FGF23) causes hypophosphatemia and consequent rickets, skeletal deformities, and growth impairment. The efficacy and safety of burosumab, a fully human monoclonal antibody against FGF23, was evaluated in two Phase 2 trials in children with XLH. In CL201, 52 children with XLH (5–12 years old, Tanner ≤2) were randomized 1:1 to receive subcutaneous burosumab every 2 (Q2W) or 4 (Q4W) weeks, with doses titrated up to 2 mg/kg to...

hrp0086p1-p479 | Fat Metabolism and Obesity P1 | ESPE2016

Hepatic Steatosis and Cardiovascular Risk in Overweight Children: Preliminary Results of the Study EFIGRO

Diez-Lopez Ignacio , Sarasua Ainhoa , Medrano Maria , Arenaza Lyde , Rodriguez-Vigil Beatriz , Tobalina Ignacio , Larrarte Eider , Perez-Asenjo Javier , Labayen Idoia

Introduction: The accumulation of fat in the liver is a serious complication of childhood obesity seems to be associated with cardiometabolic risk.Objective: To examine the association between fat accumulation in the liver and cardiovascular risk factors in overweight children.Material and methods: In the study 68 children/as (51.4% girls) were overweight or obese (defined according to the classification criteria of the Internation...

hrp0082p2-d2-549 | Puberty and Neuroendocrinology (1) | ESPE2014

Evaluation of Age at GnRH Analogue Treatment Discontinuation, Age at Menarche and Adult Height in Girls with Central Precocious Puberty from the Spanish Registry

Corripio Raquel , Soriano-Guillen Leandro , Canete Ramon , Castro-Feijoo Lidia , Escribano Arancha , Espino Rafael , Herrero-Espinet Javier , Labarta Jose-Ignacio , Argente Jesus

Background: The Spanish Registry PUBERE was created (2007) with 53 hospitals.Objective and hypotheses: i) To determine the chronological age (CA) and bone (BA) at GnRH analogue withdrawal; ii) to analyze the age at menarche and time elapsed after stopping treatment; and iii) to know adult height data.Method: Patients with central precocious puberty (CPP) born after 1992, diagnosed before 8 years in girls, with BA/CA >1 year and...

hrp0095p1-522 | Growth and Syndromes | ESPE2022

Positive impact of the TuiTek® patient support programme on caregivers of patients receiving treatment for growth hormone deficiency in Argentina

Sordelli Micaela , Debicki Matías , Gonzalez Judit , María Sosa Ana , Hernán Cejas Leopoldo , Ginaca Romina , Jheeta Amrit , Malik Sumaira , Koledova Ekaterina , Assefi Aria

Background: Good adherence to recombinant human growth hormone (r-hGH) is crucial to achieve optimal growth outcomes in children with GH deficiency (GHD). Controlling factors that negatively impact adherence is difficult in clinical practice. In order to obtain a better understanding of caregiver behaviour, an Argentinian patient support programme (PSP) implemented TuiTek®, a personalised programme comprising behavioural change training, call guides...

hrp0095p1-356 | Pituitary, Neuroendocrinology and Puberty | ESPE2022

Use of The Arginine-Stimulated Copeptin Test in Polyuric Syndrome in Paediatrics. Experience in Three Patients

Gonzalez-Llorens Nuria , Biagetti Biagetti Betina , Mogas Viñals Eduard , Puerto Carranza Elsa , Tomasini Rosangela , Campos Martorell Ariadna , Yeste Fernandez Diego , Clemente Leon Maria

Introduction: Differential diagnosis of partial central diabetes insipidus (PCDI) and primary polydipsia (PP) can be challenging. Copeptin is the C-terminal segment of the vasopressin precursor peptide that represents a novel and stable biomarker. Arginine infusion produces a nonosmotic stimulus that causes an increase in copeptin concentrations in healthy subjects. Arginine-stimulated copeptin concentrations have been used to differentiate between patients wi...

hrp0095p2-144 | GH and IGFs | ESPE2022

Growpati Study: Clinical and genetic characterization of a cohort of patients with short stature due to severe primary IGF1 deficiency

Stoupa Athanasia , Flechtner Isabelle , Viaud Magali , Pinto Graziella , Samara-Boustani Dinane , Gonzalez-Briceno Laura , Thalassinos Caroline , Amselem Serge , Legendre Marie , Netchine Irene , Brioude Frederic , Polak Michel

Background: Severe primary insulin-growth factor-1 (IGF1) deficiency (SPIGF1D) is a rare cause of short stature. Diagnosis is based on low basal IGF1 concentration, short stature, normal or elevated growth hormone concentrations and absence of any secondary causes of growth failure. Thanks to advances in next-generation sequencing (NGS) technologies, genetic etiology of SPIGF1D is expanding.Objectives: • Identify th...

hrp0092p2-128 | Fat, Metabolism and Obesity | ESPE2019

Is one year of Diet and Physical Activity Program for Obese Children Enough to Revert the Metabolic Disorders?

Garcia Cuartero Beatriz , Garcia Lacalle Concepcion , Sanchez Escudero Veronica , Sanchez Salado Laura , Martin Garcia Maria , Ara Royo Ignacio , Gonzalez Vergaz Amparo

Objetives: To analyse clinical and laboratory characteristics of an obese pediatric population.To assess their response to specific program consisting in modification of their nutritional habits and physical activity .Material and Methods: Obese patients completed a year of group therapy with nutritional education and physical activity. We analysed at the beggining and after a year anthropometry an...

hrp0089p3-p331 | Sex Differentiation, Gonads and Gynaecology or Sex Endocrinology P3 | ESPE2018

GnRH Analogues and Cross-Sex Hormonal therapy: Side Effects in Transgender Youth

Palma Cristina Mora , Fernandez Julio Guerrero , Martin Nerea Itza , Villalobos Arancha Ortiz , Bonis Ana Coral Barreda , Fresno Luis Salamanca , Casado Isabel Gonzalez

Background: Transsexuality during childhood/adolescence is a complex condition usually ending in dysphoria (GD). The prevalence of transgenderism is increasing in Pediatrics. In the process of sexual reassignment, a correct pharmacological treatment and the knowledge of possible consequences are necessary.Objetive: The objective of this study is to present the evolution of the physical and analytical characteristics and side effects in Transgender childr...