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...

hrp0084p2-412 | GH & IGF | ESPE2015

Final Height and Safety Outcomes in GH-Treated Children Born Small for Gestational Age: Experience from the Prospective GeNeSIS Observational Study

Child Chris , Quigley Charmian , Zimmermann Alan , Deal Cheri , Ross Judith , Schonau Eckhard , Blum Werner

Background: GH treatment in children born small for gestational age (SGA) has both short- and long-term growth-promoting effects and is approved in Europe and the USA (recommended dosages 0.25–0.47 mg/kg per week).Objective and hypotheses: To assess final height (FH) and safety outcomes in SGA patients receiving GH treatment in routine clinical practice using data from GeNeSIS.Method: 1208 GH-treated SGA patients were enrolled...

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...

hrp0082pl6 | Genetics of Obesity | ESPE2014

Making a Diagnosis in Severe Complex Obesity

Farooqi Sadaf

With the rising prevalence of childhood obesity, there has been an increase in the number of children presenting with severe obesity. Whilst only a relatively small proportion of severely obese children will have the classical features associated with the well-established genetic obesity syndromes such as Prader-Willi syndrome, there is increasing recognition that highly penetrant genetic disorders can frequently present as severe obesity alone without developmental delay, dys...

hrp0082s7.1 | Controversies in the Surgical Management of DSD | ESPE2014

Evolution of Feminising Genitoplasty

Pippi-Salle J L

Great controversy exists in regard to the timing and technical alternatives to perform feminizing genitoplasty in children. Opponents to an early approach argue that the reconstruction can be risky in terms of clitoral/vaginal function therefore surgery should be postponed until the patient herself can sign an informed consent and be aware of potential risks as well as confirms the desire to undergo the procedures. Such negative feelings in regard to early reconstruction are b...

hrp0086wg3.3 | ESPE Turner Syndrome Working Group (TS) | ESPE2016

The Added Value of Experience Based Coaching and the Outcomes for Women with Turner Syndrome in the Netherlands

Mijnarends Helen

To support people with chronic diseases in labour, reïntegration or participation, the Dutch Centre of Chronic Illness and Work developed a certification programme for professional experienced based coaching for patient support organisations. This 8-month during programme combines professional aid and peer-support for a diversity of chronic diseases. Experience based coaching is innovative and can assist patients emotionally by supporting them with coping and accepting th...

hrp0086p2-p317 | Diabetes P2 | ESPE2016

Type 1 Diabetes in Childhood: An 8-year Experience

Vaggopoulou Chari , Oikonomakou Maria-Zoi , Katsikareli Evanthia , Vallianatou Markella , Krokidas Georgos , Iliopoulou Maria

Background: Type 1 diabetes mellitus (DMI) is a chronic disease that requires frequent visits in outpatient pediatric endocrine clinics in order to rearrange their new lives.Objective and hypotheses: A retrospective study of patients with DMI was performed during 2008–2015, assessing epidemiological and clinical data, treatment and subsequent course.Method: We analyzed the incidence, age and gender of 75 patients at the diseas...

hrp0082p2-d2-608 | Thyroid (1) | ESPE2014

Iodine Nutrition and Neonatal Thyrotropin Levels in Lucknow, India

Bhatia Vijayalakshmi , Gopalakrishnan Vignesh , Joshi Kriti , Phadke Shubha , Dabadghao Preeti , Agarwal Meenal , Mootheden Cynthia , Das Vinita , Pandey Amita

Introduction: In India, newborn screening (NBS) for congenital hypothyroidism (CH) is not yet a national government policy. Preliminary information suggests neonatal TSH levels to be higher in expatriate Indian babies than those in European populations in developed countries.Objectives: To evaluate maternal urinary iodine and cord blood TSH in a subset of women participating in a postnatal NBS program for CH.Methods: Cord blood (CB...