hrp0082p2-d1-414 | Growth Hormone | ESPE2014

Final Height SDS Gain of GH Treated Children with SHOX Deficiency Describing Observational and Clinical Trial Data

Benabbad Imane , Child Christopher , Carel Jean-Claude , Rosilio Myriam

Background: Patients with mutations of the short-stature-homeobox-containing (SHOX) gene likely have impaired growth, with or without a spectrum of skeletal anomalies consistent with mesomelic skeletal dysplasia. In a multinational clinical trial, GH has been shown to increase growth rate and final height (FH).Objective and hypotheses: The aim of this analysis was to describe FH outcome after GH treatment in an observational setting (Genetics and Neuroen...

hrp0089rfc13.2 | Pituitary, Neuroendocrinology and Puberty 2 | ESPE2018

Growth Outcomes and Near Adult Height of Children with Congenital GH Deficiency (GHD) due to Abnormal Pituitary Development: Data from a Prospective, Multinational Observational Study

Child Christopher , Leger Juliane , Deal Cheri , Benabbad Imane , Jia Nan , Blum Werner

Background: Children with structural hypothalamic-pituitary anomalies, e.g. ectopic posterior pituitary (EPP) with/without pituitary stalk interruption syndrome, septo-optic dysplasia (SOD), and isolated anterior pituitary aplasia/hypoplasia (AP/HP) usually have more severe GHD and better auxological outcomes with GH therapy than those with normal hypothalamic-pituitary magnetic resonance imaging findings. However, adult height data is limited.Objective:...

hrp0082p1-d3-160 | Growth (2) | ESPE2014

Final Height and Safety Outcomes in GH-Treated Children with Short Stature Homeobox-Containing Gene (SHOX) Deficiency: Experience From a Large, Multinational, and Prospective Observational Study

Child Christopher , Quigley Charmian , Zimmermann Alan , Ross Judith , Deal Cheri , Drop Stenvert , Blum Werner

Background: To date, one randomized, controlled, clinical trial (RCCT) demonstrated that GH-treated patients with SHOX deficiency (SHOX-D) had GH-mediated height gain comparable to that of girls with Turner syndrome (TS). No new safety concerns were identified, but the study was limited by small sample size.Objective and hypotheses: To examine long-term outcomes in patients treated in standard clinical practice, we assessed final height (FH) and safety o...

hrp0084p1-82 | Growth Hormone | ESPE2015

Assessment of Primary Cancers in Growth Hormone–Treated Paediatric Patients Compared with General Population Registries: An Epidemiological Analysis of a Large, Multinational, Prospective Observational Study

Child Christopher , Zimmermann Alan , Jia Nan , Robison Leslie , Bramswig Jurgen , Blum Werner

Background: Concern remains regarding the potential influence of growth hormone (GH) treatment on neoplasia because of the general growth-inducing effect of GH and associations between high serum IGF1 concentrations and certain cancers in adulthood. Many studies that evaluated risk for primary cancer in GH-treated patients without previous malignancy found no increased rates of primary neoplasia. A higher risk for colorectal cancer was observed in a single-country cohort treat...

hrp0086p1-p628 | Growth P1 | ESPE2016

Height Gain and Safety Outcomes in Growth Hormone (GH)-Treated Girls and Boys with Idiopathic Short Stature (ISS): Experience from the Prospective GeNeSIS Observational Study

Child Christopher , Quigley Charmian , Zimmermann Alan , Deal Cheri , Ross Judith , Rosenfeld Ron , Cutler Jr Gordon , Blum Werner

Background: GH treatment for ISS received first approval in the USA in 2003 based on data from two controlled clinical trials. Eligibility is restricted to those with baseline (BL) height standard deviation score (HtSDS) ≤−2.25; other approvals followed, but not in Europe.Objective and hypotheses: To assess outcomes of GH therapy in a large cohort of patients (pts) treated in routine clinical practice.Methods: Short-ter...

hrp0086rfc15.6 | Late Breaking | ESPE2016

Safety of GH in Paediatrics: The GeNeSIS Prospective Observational Study Experience between 1999 and 2015 (NCT01088412)

Blum Werner , Child Christopher , Chrousos George , Cummings Elisabeth , Deal Cheri , Hasegawa Tomonobu , Holterhus Paul-Martin , Jia Nan , Lawrence Sarah , Linglart Agnes , Loche Sandro , Maghnie Mohamad , Sanchez Jacobo Perez , Polak Michel , Predieri Barbara , Richter-Unruh Annette , Rosenfeld Ron , Tajima Toshihiro , Yeste Diego , Yorifuji Tohru

Background: Although GH’s safety profile since 1987 is good, concerns remain regarding cancer (CA) risk, and French SAGhE data indicated increased mortality and cerebrovascular disease (CVD) in certain GH-treated patients (pts).Objective and hypotheses: To evaluate key safety outcome incidence in GH-treated pts of all short stature diagnoses (dx) who participated in GeNeSIS (1999–2015, 30 countries).Methods: Pt history/ca...

hrp0089p1-p181 | Growth & Syndromes P1 | ESPE2018

Long Term Effects of Childhood Growth Hormone Treatment on Height and Body Mass Index in Adolescents and Adults with Silver-Russell Syndrome

Lokulo-Sodipe Oluwakemi , Canton Ana P. M. , Giabicani Eloise , Ferrand Nawfel , Child Jenny , Wakeling Emma L. , Binder Gerhard , Netchine Irene , Mackay Deborah J.G. , Inskip Hazel M. , Byrne Christopher D. , Davies Justin H. , Temple I. Karen

Growth hormone (GH) is commonly used during childhood to treat short stature in Silver-Russell syndrome (SRS), but final height and long-term body mass index (BMI) data are limited.Objective: To evaluate height and BMI in older individuals with molecularly confirmed SRS and compare those previously treated with GH to those untreated.Methods: Growth data on individuals aged ≥13 years with SRS were evaluated from UK, French and...

hrp0092p3-253 | Thyroid | ESPE2019

Child Thyrotoxicosis Syndrome: Structure and Characteristics

Evsyukova Evgeniya , Kolomina Irina , Bukin Sergey , Kisileva Elena , Latyshev Oleg , Okminyan Goar , Samsonova Lyubov

Objective: To study the structure and characteristics of child thyrotoxicosis syndrome.Materials and Methods: At the 1st stage, medical records of patients treated at the Endocrinology Unit of the Children's Hospital named after Z.A. Bashlyaeva of the City of Moscow in 2014-2018 (n=4530) were analyzed. At the 2nd stage, a primary examination of 106 children 3 to 17 years old with a diagnosis of thyrotoxi...

hrp0089p3-p314 | Pituitary, Neuroendocrinology and Puberty P3 | ESPE2018

Klinefelter Syndrome with Ambiguous Genitalia in a Child

Faizi Muhammad , Rochmah Nur , Purwana Arie

Background: Klinefelter Syndrome (KS) is the most common sex chromosome disorder in males caused by additional X chromosome. It is characterized by progressive testicular failure. KS patient usually have complete male sexual differentiation without genital ambiguity. The prevalence of KS is 1 in 660 males which only 10% are detected before or during puberty, and about two third so fall men with X-chromosome polyploidies fail to be identified during their lifetime.<p class=...

hrp0094p2-478 | Thyroid | ESPE2021

A child with anti-thyroid arthritis syndrome

Kilci Fatih , Sönmez Hafize Emine , Jones Jeremy , Közler Selen Hürmüzlü , Çizmecioğlu Jones Filiz Mine

Introduction: Anti-thyroid arthritis syndrome (AAS) is a rare complication that may occur in patients treated with anti-thyroid drugs. Presentation is variable and may include myalgia, arthralgia, skin rash, high fever, and polyarthritis. Herein, we present a girl diagnosed with AAS. Case report: A 9-year-old girl was admitted to emergency complaining of fever and arthralgia. She was the second child of non-consanguineous healthy parents. She had been d...