Previous issue | Volume 86 | ESPE2016 | Next issue

55th Annual ESPE

Paris, France
10 Sep 2016 - 12 Sep 2016

Card image cap
Paris, France: 10-12 September 2016 Further information

hrp0086p1-p593 | Growth P1 | ESPE2016

The Mechanistic Role of Fibroblast Growth Factor 21 (FGF21) in Growth Hormone Resistance Secondary to Chronic Childhood Conditions

Mistry Jayna , Ruiz-Babot Gerard , Guasti Leonardo , Dunkel Leo

Background: Both undernutrition and chronic inflammation impair linear growth through resistance to GH. Fibroblast growth factor 21 (FGF21) is known as an important regulator of the metabolic adaptation to fasting. Elevated expression of FGF21, secondary to prolonged undernutrition has been identified to develop GH resistance and subsequent attenuation of skeletal growth and growth plate chondrogenesis in both mice and human. However, the mechanism of FGF21&#1...

hrp0086p1-p594 | Growth P1 | ESPE2016

Somapacitan, a Once-Weekly Reversible Albumin-Binding Growth Hormone (GH) Derivative, Is Well Tolerated and Convenient in Adults with GH Deficiency (AGHD): Results from a 26-Week Randomised, Controlled Phase 3 Trial

Johannsson Gudmunder , Feldt-Rasmussen Ulla , Holme Haakonsson Ida , Biering Henrik , Rodien Patrice , Tahara Shigeyuki , Toogood Andrew , Hojby Michael

Background: Growth hormone (GH) replacement as daily s.c. injections for patients with adults with GH deficiency (AGHD) can be cumbersome. Somapacitan (Novo Nordisk), a once-weekly reversible albumin-binding GH derivative, has been shown in short-term trials to be well tolerated in healthy adults and in patients with AGHD.Objective and hypotheses: This trial was a multinational, multicentre, randomised (2:1), open-label, active-controlle...

hrp0086p1-p595 | Growth P1 | ESPE2016

The Diagnostic Value of IGF-II, IGF-I and IGFBP-3 in Silver–Russell Syndrome

Binder Gerhard , Eggermann Thomas , Weber Karin , Schweizer Roland

Background: Recently we described a family with several members having intrauterine and postnatal growth failure as well as signs of Silver–Russell syndrome (SRS) who carried a heterozygote nonsense mutation of IGF2. The patients had low IGF-II serum levels, but normal IGF-I serum levels.Objective and hypotheses: We aimed to estimate the diagnostic value of the IGF-II, IGF-I and IGFBP-3 measurements in the assessment of children with SRS.<p clas...

hrp0086p1-p596 | Growth P1 | ESPE2016

Functional in vitro Characterization of Two Novel Germinal STAT3 Mutations Associated with Short Stature, Immunodeficiency and Autoimmune Disease

Gutierrez Mariana , Scaglia Paula , Keselman Ana , Martucci Lucia , Karabatas Liliana , Domene Sabina , Blanco Miguel , Sanguinetti Nora , Bezrodnik Liliana , Di Giovanni Daniela , Caldirola Soledad , Esnaola Azcoiti Maria , Jones Nana-Hawa , Hwa Vivian , Revale Santiago , Vazquez Martin , Jasper Hector , Kumar Ashish , Domene Horacio

Background: We have recently reported the molecular diagnosis of two patients with severe growth failure associated with a spectrum of early-onset autoimmune disease and immunodeficiency. Heterozygous de novo mutations, c.1847_1849delAAG (p.E616del) and c.1276T>C (p.C426R), in the STAT3 gene were found. Functional in vitro studies of these variants are presented.Objective and hypotheses: We...

hrp0086p1-p597 | Growth P1 | ESPE2016

Abstract withdrawn...

hrp0086p1-p598 | Growth P1 | ESPE2016

Human Phase1 Clinical Data of ALT-P1 (hGH-NexP) by Healthy Korean Males

Mee Lee Sang , Cho Jung-Soo , Shin Chung Hye , Soo Park Min , Jae Park Soon

Background: ALT-P1 (CJ-40002) is a long-acting recombinant growth hormone (GH) fused with NexP, which is a long-acting carrier developed by Alteogen Inc. NexP is a protein engineered recombinant alpha1 antitrypsin with further increased in vivo half-life without a native proteinase inhibitor activity. In non-clinical studies of cynomologus monkeys, the extended half-life of hGH-NexP has been successfully proved without side effects in high dose as 20 mg per kg dose.<p clas...

hrp0086p1-p599 | Growth P1 | ESPE2016

Validation of Prediction Models for Near Final Adult Height in Children with Idiopathic Growth Hormone Deficiency Treated with Growth Hormone for 1 Year

Straetemans Saartje , De Schepper Jean , Thomas Muriel , Verlinde Franciska , Rooman Raoul

Background: An accurate prediction of final height after the first year of growth hormone (GH) treatment may help clinicians to give parents and children more realistic expectations.Objective and hypotheses: To validate two prediction models (with and without max. GH peak) for near final adult height (nFAH) by Ranke et al.Method: Height data of 142 (93 male) idiopathic GH deficient (iGHD) children, treated with GH for at l...

hrp0086p1-p600 | Growth P1 | ESPE2016

The Influence of Recombinant Human Growth Hormone Treatment on Very Small Embryonic/Epiblast Like Stem Cells

Wedrychowicz Anna , Sielatycka Katarzyna , Kubis Ewa , Roztoczynska Dorota , Starzyk Jerzy B. , Ratajczak Mariusz Z.

Background: Present knowledge on the effects of growth hormone (GH) on aging and lifespan are controversial. Clinical data indicate that normal or high levels of GH may accelerate aging and increase the risk of cardio-vascular diseases. Very small embryonic-like stem cells (VSELs) are a population of developmentally early stem cells residing in adult tissues, which could have the potential role in aging and organ rejuvenation.Objective: The aim of the st...

hrp0086p1-p601 | Growth P1 | ESPE2016

A Lipid-Based System for the Oral Delivery of Growth Hormone

Fricker Gert , Pantze Silvia , Parmentier Johannes , Helm Frieder , Hartmann Klaus , Gropp Felix

Background: Bioavailability of peptide/protein – drugs is extremely low after oral administration due to their instability in the gastrointestinal tract or poor absorption.Objective and hypotheses: Oral delivery of growth hormone and somatostatin by adding extreme stable lipids.Method: A liposomal system based on a combination of standard lipids and membrane spanning tetraether lipids, which are extremely stable biomolecules. ...

hrp0086p1-p602 | Growth P1 | ESPE2016

Prediction of First Year Response to Growth Hormone Treatment in Neural Network Models

Smyczynska Urszula , Smyczynska Joanna , Hilczer Maciej

Background: Accurate prediction of responsiveness to growth hormone (GH) therapy is an important issue. The 1st year response to treatment is regarded as significant predictor of the attained final height. Neural networks are techniques of machine learning which do not require any assumptions and preprocessing of data, contrary to linear regression models.Objective and hypotheses: The aim of the study was to predict height velocity (HV) during 1st year o...

hrp0086p1-p603 | Growth P1 | ESPE2016

Pediatric Phase 2 Data Demonstrate that TransCon hGH Has an Anti-hGH Immunogenic Profile that is Comparable to Daily hGH

Gilfoyle David , Pihl Susanne , Chatelain Pierre , Beckert Michael

Background: TransCon hGH, is a once-weekly prodrug releasing unmodified hGH, for the treatment of Growth Hormone Deficiency (GHD) in children and adults. To date, TransCon hGH has demonstrated comparable efficacy, safety and anti-hGH immunogenic profile to daily hGH, with no occurrence of neutralizing antibodies.Objective and hypotheses: Protein-based therapies may be associated with generation of drug-specific antibodies, which may impact efficacy, part...

hrp0086p1-p604 | Growth P1 | ESPE2016

The Exon3-Deleted Growth Hormone Receptor Gene Polymorphism (d3-GHR) is Associated with Increased Spontaneous Growth and Impaired Insulin Sensitivity in Prepubertal Short SGA Children (NESGAS)

Wegmann Mathilde Gersel , Jensen Rikke Beck , Thankamony Ajay , Kirk Jeremy , Donaldson Malcolm , Ivarsson Sten-A , Soder Olle , Roche Edna , Hoey Hillary , Dunger David B , Juul Anders

Background: A common polymorphism in the growth hormone receptor gene (d3-GHR) was found to be associated with pre- and postnatal growth and GH-induced growth. D3-GHR was associated with glucose metabolism in adults with GHD and acromegaly, but this has not previously been explored in children.Objective and hypotheses: We examined the impact of the GHR-exon-3 deletion on glucose metabolism and anthropometrics in short SGA children before and following 1 ...

hrp0086p1-p605 | Growth P1 | ESPE2016

A New Case of Intragenic Deletion in IGF1R with Very Mild Phenotype

Coppola Ruggero , Luongo Caterina , Nacca Raffaella , Sasso Marcella , Grandone Anna , del Giudice Emanuele Miraglia , Perrone Laura

Background: IGF1R mutations are characterized by IGF-1 resistance causing impaired fetal and postnatal growth. Several reports in children with heterozygous defects of IGF1R have demonstrated a variable phenotype, which can be associated to microcephaly, dismorphic features and mild developmental delay.Case presentation: We report of an 8-years-old boy, who came at our observation with short stature (−3.2 SDS) and mild microcephal...

hrp0086p1-p606 | Growth P1 | ESPE2016

The Role of IGF-1R Gene Polymorphisms with Regard to Susceptibility to Idiopathic Short Stature Risk in the Chinese Population of Jiangxi Area

Yang Yu , Huang Hui , Yu Zhen , Wang Wei , Yang Li , Huang Wei , Xie Liling

Background: Accumulated evidence indicates that the GH-IGF-1 pathway might be one of the crucial mechanisms of ISS. Insulin-like growth factor-1 receptor (IGF-1R) is the effector molecule that regulates the cascade reaction of hormone receptors in the GH–IGF-1 axis.Objective and hypotheses: To investigate the role of IGF-1R gene polymorphisms with regard to susceptibility to Idiopathic short stature risk in the Chinese population of Jiangxi area.</p...

hrp0086p1-p607 | Growth P1 | ESPE2016

Effects of the Addition of Metformin to Recombinant Human GH on Bone Maturation and Pubertal Progression in Short Children Born Small-for-Gestational-Age

Capistros Mireia Tirado , Sancho Paula Casano , Toda Lourdes Ibanez

Background: Small for gestational age (SGA) children who experience rapid and exaggerated postnatal catch-up are prone to develop insulin resistance and to progress faster into puberty, resulting in a shorter final height. Short, non-catch-up SGA children treated with recombinant human GH (rhGH) may present with the same sequence. In a previous controlled study from our group performed in short SGA children, metformin – added to rhGH therapy – improved endocrine-meta...

hrp0086p1-p608 | Growth P1 | ESPE2016

Pubertal Height Gain in Females with Isolated Growth Hormone Deficiency Treated with rhGH Alone or in Combination with GnRHan

Voutetakis Antonis , Chiotis Dimitris , Gryparis Alexandros , Kanaka-Gantenbein Christina , Dacou-Voutetakis Catherine

Background: A significant component of total linear growth is height gain achieved after the initiation of puberty. Children with Isolated GH Deficiency (IGHD) frequently come for evaluation around the peripubertal stage. Therefore, increasing pubertal height gain in IGHD children entering puberty with a relatively low height is important. Researchers have tried to assess effectiveness of treatment in such patients based on various key measurements and a variety of end-points....

hrp0086p1-p609 | Growth P1 | ESPE2016

Cognitive Abilities and Academic Achievement Among Youths with Short Stature Receiving Growth Hormone Therapy

Yeguez Carlos , Gardner Melissa , Sandberg David

Background: Reports suggest that youths with short stature (SS) exhibit academic under-achievement relative to cognitive aptitude and GH treatment diminishes the difference. However, interpretation of this achievement-aptitude discrepancy is confounded by the use of achievement and intelligence tests normed in different samples.Objective and hypotheses: To assess whether reports of academic underachievement in SS samples are partially attributable to cho...

hrp0086p1-p610 | Growth P1 | ESPE2016

One Year Use of Anastrazole Improves the Predicted Adult Height of Male Adolescents with and without Associated GH Therapy

Pinto Renata Machado , Goncalves Macks Wendhell

Background: Estrogen is an essential regulator of bone maturation, growth plate fusion, and cessation of longitudinal growth. Aromatase inhibitors (AI) block the conversion of androgens to estrogens, and can be used to delay bone maturation in males.Objective and hypotheses: We sought to determine whether the blockage of estrogen biosynthesis due to the use of the AI Anastrazole increases the Predicted Adult Height (PAH) in boys with short stature.<p...

hrp0086p1-p611 | Growth P1 | ESPE2016

IGFALS Gene Deletion in a Family with Short Stature

Haliloglu Belma , Losekoot Monique , Kaya Avni , Wit Jan-Marteen

Background: ALS deficiency is characterized by mild short stature, delayed puberty, low serum IGF1, low serum IGFBP3 and undetectable serum ALS levels.Case: A 11.3 years old boy presented with short stature. He was born at term to consanguineous parents and the birth weight was unknown. On physical examination, his height and weight were 130.5 cm (−2.33 SDS) and 25.2 kg (−2.36 SDS) and he was prepubertal. The routine laboratory tests were nor...

hrp0086p1-p612 | Growth P1 | ESPE2016

Adherence with Twice-Monthly, At-Home Dosing Schedule of Somavaratan (VRS-317) Long-Acting Growth Hormone Treatment in Children with Growth Hormone Deficiency (GHD) (NCT02068521)

Humphriss Eric , Sheikh F. Naureen , Seaman Morgan , Ng David , Bright George

Background: Treatment adherence to daily subcutaneous rhGH is a burden for GHD patients, with noncompliance reported in up to 77% of patients and significantly associated with reduced efficacy (Rosenfeld Endocr Pract 2008; Cutfield PLoS One 2011). Somavaratan, a novel rhGH fusion protein with t1/2 >100 h, demonstrated clinically meaningful improvements in height velocity and IGF-I in prepubertal children with GH deficiency (GHD) in a multicenter, randomized, Pha...

hrp0086p1-p613 | Growth P1 | ESPE2016

Glucose Dysregulation in Children with Growth Hormone Deficiency (GHD), Turner Syndrome (TS) or Born Small for Gestational Age (SGA) Treated with GH: A Report from the NordiNet International Outcome Study (IOS)

Kotnik Primoz , Rohrer Tilman , Pedersen Birgitte Tonnes , Pournara Effie , Christesen Henrik

Background: The prevalence of glucose dysregulation in children treated with GH is not well established.Objective and hypotheses: To evaluate the prevalence of glucose dysregulation in children with growth disorders (GH deficiency (GHD), Turner syndrome (TS), small for gestational age (SGA)) treated with GH (Norditropin, Novo Nordisk) enrolled in NordiNet International Outcome Study (IOS) (NCT00960128), a non-interventional study evaluating safety and ef...

hrp0086p1-p614 | Growth P1 | ESPE2016

Serum α-Klotho Levels are not Informative for the Evaluation of GH Secretion in Short Children

Elsedfy Heba , Meazza Cristina , Radetti Giorgio , Khalaf Randa I. , Pagani Sara , Sessa Nicodemo , Albertini Riccardo , De Stefano Anna Maria , Navarra Antonella , Lupi Fiorenzo , El Kholy Mohamed , Bozzola Mauro

Background: α-klotho is a transmembrane protein which can be cleaved and act as a circulating hormone. Since low α-klotho levels were found in organic GH deficiency (GHD) and high levels in acromegaly, an interaction between α-klotho, GH and linear growth has been suggested.Objective and hypotheses: We investigated the role of α-klotho protein as a reliable marker of GH secretion in short children and the factors influencing its secre...

hrp0086p1-p615 | Growth P1 | ESPE2016

Immunogenicity Results of Once-Weekly Administration of CTP-Modified Human Growth Hormone (MOD-4023): A Phase 2 Study in Children with Growth Hormone Deficiency

Mendelson Michal Jaron , Bar-Ilan Ahuva , Hershkovitz Oren , Hart Gili

Background: CTP-modified hGH (MOD-4023) has been developed for once weekly administration in GH deficient (GHD) adults and children. Immunogenicity samples of once-weekly s.c. administration of MOD-4023 were detected for the presence of binding and neutralizing anti-MOD-4023 Ab’s in pediatric Phase 2 study.Objective and hypotheses: During the first year of the study, 53 pre-pubertal GHD children were treated with once-weekly s.c. injections of one o...

hrp0086p1-p616 | Growth P1 | ESPE2016

Serum IGF-I, IGFBP-3 and Als Concentrations and Physical Performance in Young Swimers During a Training Season

Filho Hugo Tourinho , Pires Marcela , Puggia E F , Papoti M , Barbieri R , Martinelli Jr Carlos

Background: Exercise programs are related to the anabolic function of GH/IGF-I axis.Objective and hypotheses: To analyse IGF-I, IGFBP-3 and ALS serum concentrations in adolescent swimmers at different stages of training season, and compare them with physical performance and body composition.Method: Nine male athletes, aged 16–19 years, who trained regularly throughout the season, were studied. IGF-I, IGFBP-3 and ALS were recor...

hrp0086p1-p617 | Growth P1 | ESPE2016

The Blood Antioxidant System in Adult Growth Hormone Deficient Patients after Concluded Childhood Growth Hormone Therapy

Vorontsova Maria , Pankratova Maria , Yusipovich Alexander , Adil Baizhumanov , Shiryaeva Tatyana , Nagaeva Elena , Georgiy Maximov , Peterkova Valentina

Background: The antioxidant system that protects tissues from damaging oxidation processes is a universal indicator for metabolic balance. It is known that GH deficiency (GHD) is associated with a high risk of developing metabolic disorders.Objective and hypotheses: The aim of this study was to examine the effects of inadequate GH secretion on the markers of the blood antioxidant system in adult GHD patients.Method: The study inclu...

hrp0086p1-p618 | Growth P1 | ESPE2016

Design and Clinical Development of TransCon Growth Hormone for Growth Hormone Deficiency (GHD)

Beckert Michael , Gilfoyle David , Mikkelsen Jan Moller , Rasmussen Grethe , Rau Harald , Sprogoe Kennett

Background: TransCon GH is designed as a once-weekly sustained-release prodrug of recombinant human GH (hGH, somatropin). Based on the inert TransCon prodrug technology unmodified native hGH is released with a Cmax and AUC comparable to daily therapy. TransCon GH leverages the known pharmacology of daily hGH and is being developed for the treatment of GH deficiency (GHD) in children and adults.Objective and hypotheses: Develop a safe and efficacious sust...

hrp0086p1-p619 | Growth P1 | ESPE2016

Mutations in PROP1 Gene in Combination with 47,XYY Karyotype: Case Report

Pankratova Maria , Gubaeva Diliara , Kareva Maria , Tiulpakov Anatoly , Peterkova Valentina

Background: Mutations in PROP1 gene are the most common known genetic cause of multiple pituritary hormone deficiency. It is characterized by somatolactotroph, thyrotroph, gonadotroph and sometimes corticotroph deficiencies and pituitary hyper- or hypoplasia. The karyotype 47,XYY occurrs in 1 in every 1000 live male birth. Some studies report that the phenotype of XYY syndrome includes tall stature, behavioral problems and low fertility.Objectiv...

hrp0086p1-p620 | Growth P1 | ESPE2016

Efficacy of Growth Hormone Treatment in Patients with Type 1 Diabetes Mellitus and Growth Hormone Deficiency

Bonfig Walter , Lindberg Anders , Cutfield Wayne , Dunger David , Camacho-Hubner Cecilia , Holl Reinhard W

Background: The combination of type 1 diabetes mellitus (T1DM) and GH deficiency is uncommon. In a previous study (1) we found that in children with T1DM and GHD with adequate adaptation of insulin dosage, the metabolic control of T1DM did not worsen during GH treatment. However, decreased catch-up growth was observed and no data on GH dose was available.Objective: To analyse first year treatment growth response and GH dosage in prepubertal patients with...

hrp0086p1-p621 | Growth P1 | ESPE2016

Final Height after Growth Hormone Treatment in Children with Chronic Renal Failure

Lonero Antonella , Bizzarri Carla , Delvecchio Maurizio , Emma Francesco , Strologo Luca Dello , Cappa Marco

Background: Growth retardation is seen in about 30% of children with chronic renal failure (CRF). Under-nutrition, anaemia, secondary hyperparathyroidism, acidosis, corticosteroid therapy and abnormalities in the GH/insulin like growth factor system have been implicated. Recombinant GH (rGH) therapy is recommended in children showing failure to maintain a normal height velocity despite optimized primary treatments.Objective and hypotheses: It has been de...

hrp0086p1-p622 | Growth P1 | ESPE2016

Major Plasma Carotenoids Levels in Growth Hormone Deficient Children

Pankratova Maria , Yusipovich Alexander , Vorontsova Maria , Baizhumanov Adil , Cherkashin Alexander , Shiryaeva Tatiana , Solovchenko Alexei , Peterkova Valentina

Background: Carotenoids are potent antioxidants that affect many different metabolic processes. In plasma, carotenoids are transported with lipoproteins. Growth hormone deficiency (GHD) is known to induce oxidative stress and deterioration in the lipid profile, which can change the level and composition of carotenoids. Particularly interesting to measure these parameters in GHD children.Objective and hypotheses: The aim of this study is to examine the am...

hrp0086p1-p623 | Growth P1 | ESPE2016

Autosomal Dominant Growth Hormone Deficiency due to a Novel Mutation in the gh1 Gene

Ternand Christine , Gao Harry , Miller Bradley

Background: Familial growth hormone deficiency (GHD) with an autosomal dominant inheritance pattern (isolated GHD type II) due to multiple different mutations in the GH1 gene have been described.Objective and hypotheses: Describe the clinical characteristics and mutation analysis of affected individuals in a family with growth hormone deficiency inherited in an autosomal dominant pattern.Method: Medical record review.<p class="...

hrp0086p1-p624 | Growth P1 | ESPE2016

A Novel GH1 Mutation in a Family with Autosomal-Dominant Type II Isolated Growth Hormone Deficiency

Gurbuz Fatih , Elmaogullari Selin , Arasli Aslihan , Demirel Fatma

Background: The familial type of isolated growth hormone deficiency (IGHD) is characterized by a variable degree of growth restriction, low but detectable GH serum concentrations. The recessive type IA and IB, the autosomal-dominant type II, and X-linked recessive type III. Phenotype-genotype correlations are notoriously difficult to be established. Herein, we described the patient who has autosomal-dominant type II IGHD due to a novel GH1 mutation.Objec...

hrp0086p1-p625 | Growth P1 | ESPE2016

2nd Year Pharmacokinetic and Pharmacodynamic Modeling of Long-Acting Human Growth Hormone (MOD 4023) in Growth Hormone Deficient Children

Fisher Dennis M. , Jaron Mendelson Michal , Vander Shelly , Koren Ronit , Hart Gili

Background: OPKO Biologics is developing MOD-4023, a long-acting growth hormone (GH), intended for weekly dosing for the treatment of idiopathic GH deficiency in children. At ESPE2015, we presented pharmacokinetic (PK) and pharmacodynamic (PD, based on IGF-1) models for weekly MOD-4023 administration in children aged 3–11 years. Those models were based on data collected during the ‘PKPD period’ (the second steady state dose of MOD-4023) and monthly values during...

hrp0086p1-p626 | Growth P1 | ESPE2016

Significance of IGF-I Generation Test in Diagnosing Primary and Non-Primary IGF-I Deficiency – Clinical Considerations

Smyczynska Joanna , Smyczynska Urszula , Stawerska Renata , Lewinski Andrzej , Hilczer Maciej

Background: The diagnosis of severe primary IGF-I deficiency (IGFD) in children with normal growth hormone (GH) peak in stimulation tests (stimGH) should be confirmed by IGF-I increase <15.0 ng/ml during generation test (IGF-GT), however the significance of IGF-GT has been questioned by some researchers.Objective and hypotheses: Evaluation of the significance of IGF-GT in children with normal stim GH and IGFD, with respect to efficacy of growth-promo...

hrp0086p1-p627 | Growth P1 | ESPE2016

Change of Growth Pattern and Thickness of Epiphyseal Plate in Female Rats According to Injected Estrogen Dosage

Kang ByungHo , Shim Kye Shik , Lim Sung Jig , Cho Ja Hyang

Objective and hypotheses: The purpose was to get the basic data of optimum serum concentration of estrogen in maximizing pubertal growth spurt, and decreasing the acceleration of epiphyseal closure of long bones.Method: i) Fifteen female SD rats (13-week aged; post pubertal growth spurt) were randomly divided into three groups. After 1 week, the group 1 were injected subcutaneously with sesame oil, as a control, group 2 were with 10 μg/kg per week o...

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...

hrp0086p1-p629 | Growth P1 | ESPE2016

Treatment of Resistant Paediatric Somatotropinomas due to AIP Mutation with Pegvisomant

Joshi Kriti , Zacharin Margaret

Background: Somatotropinomas are rare in childhood and are frequently associated with genetic mutations. AIP mutations are found in 20–25% cases of sporadic pediatric adenomas and are most commonly associated with GH secreting tumours that are large, aggressive and may be resistant to medical therapy.Objective and hypotheses: To assess response to Pegvisomant, a GH receptor antagonist in two children with sporadic somatotropinomas due to AIP mutatio...

hrp0086p1-p630 | Growth P1 | ESPE2016

GH-Pattern with High Trophs are Often Found after Daily sc rhGH-Injection in Children

Lundberg Elena , Andersson Bjorn , Kristrom Berit , Rosberg Sten , Albertsson-Wikland Kerstin

Background: Endogenous GH pattern is characterized by high peaks (growth signal) and low trophs (metabolic signal). Exogenous GH is given by subcutaneous injection (scGH-injection) daily at bedtime.Objective and hypotheses: To study the factors influencing intra-/interindividual variation of pharmacokinetics and pattern of scGH-injection in GH treated children.Method: One hundred and twenty eight subjects followed yearly ≤ 8 ...

hrp0086p1-p631 | Growth P1 | ESPE2016

Are the GH Treatment Doses in Use within Secretion Rates of Healthy Children?

Lundberg Elena , Andersson Bjorn , Kristrom Berit , Rosberg Sten , Albertsson-Wikland Kerstin

Background: GH-secretion rates for children ranges in pre/early puberty 0.1–11 U/24 h and during mid-puberty 4–40 U/24 h. This can be used to optimize the rhGH treatment doses in children.Objective and hypotheses: To calculated the bioavailable rhGH in relation to injected dose and compare this to GH-secretory rate in healthy children; and to investigate factors influencing bioavailability.Method: One hundred and twenty e...

hrp0086p1-p632 | Growth P1 | ESPE2016

The Influence of Growth Hormone Treatment on Fat-free Mass in Prepubertal Children with Kabuki Syndrome

Remmel Robin , Schott Dina , Gerver Willem-Jan , Stumpel Constance

Background: The influence of GH on prepubertal children with Kabuki Syndrome (KS) is a novel field of research. KS is a congenital anomaly/intellectual disability syndrome caused by a mutation in the KMT2D or KDM6A gene. These mutation causes distinct phenotypically features, such as short stature and facial dysmorphology. Earlier studies describe a high incidence of obesity in children with KS.Aims and objectives: In this prospective s...

hrp0086p1-p633 | Growth P1 | ESPE2016

Evaluation of Prepubertal Patients with Suspected Neurosecretory Dysfunction of Growth Hormone Secretion: Diagnostic Steps and Treatment Response

Sydlik Carmen , Weiszenbacher Claudia , Pozza Susanne Bechtold-Dalla , Schmidt Heinrich

Background and aims: Existence and diagnostic procedures of neurosecretory dysfunction (NSD) are still a matter of debate. The aim of the study was to analyse prediagnostic data of short-statured children with pathologic and normal spontaneous GH-secretion and to evaluate the effect of GH-therapy in NSD-patients.Methods: Of 90 children aged 3–16 years, in whom 12-hour night profiles for GH-secretion were performed (unicentric), in 49 NSD was diagnos...

hrp0086p1-p634 | Growth P1 | ESPE2016

Four-Year Results from PATRO Children, a Multi-Centre, Non-Interventional Study of the Long-Term Safety and Efficacy of Omnitrope® in Children Requiring Growth Hormone Treatment

Pfaffle Roland , Kanumakala Shankar , Charlotte Hoybye , Berit Kristrom , Markus Zabransky , Tadej Battelino , Michel Colle

Background: PATRO Children is an international, open, longitudinal, non-interventional study of the long-term safety and efficacy of Omnitrope®, a biosimilar recombinant human GH (rhGH).Objective and hypotheses: The primary objective of PATRO Children is to assess long-term safety of Omnitrope® (particularly the diabetogenic potential of GH in short children born small for gestational age, the risk of malignancies, and oth...

hrp0086p1-p635 | Growth P1 | ESPE2016

Glucagon vs Clonidine Stimulation for Testing Growth Hormone Secretion in Children and Adolescents: Which is Better?

Asher Galit , Diament Rotem , Phillip Moshe , Lebenthal Yael

Background: The definitive diagnosis of childhood GH deficiency (GHD) depends on the demonstration of failure to respond to two stimuli. In our center children are allocated to either glucagon-first or clonidine-first according to the preference of the pediatric endocrinologist following the patient. The nursing staff prefer glucagon-first due to patient safety (less pronounced adverse events and faster recovery time). Few studies have addressed which GH stimulation test shoul...

hrp0086p1-p636 | Growth P1 | ESPE2016

The Use of Tissue Doppler Imaging in Assessing Right and Left Ventricle Diastolic Function in Children with Growth Hormone Deficiency before and after 1-Year Therapy with Growth Hormone

Khalaf Randa , Elkholy Mohamed , Elsedfy Heba , Kotby Alyaa , Hamza Rasha , Youssef Omneya

Background: Growth Hormone (GH) therapy has a positive effect on many parameters including metabolic and physiologic functions as well as its effect on growth. It has also been shown that GH therapy exerts a significant effect on cardiac morphology and function as evidenced by echocardiographic findings.Aim: To investigate left and right ventricle (LV, RV) diastolic function by Tissue Doppler imaging (TDI) in pre-pubertal growth hormone deficient (GHD) c...

hrp0086p1-p637 | Growth P1 | ESPE2016

Diagnosing GH Deficiency in Children by Arginine Hydrochloride Infusion Test: Relationship between Auxiological Characteristics, Arginine Plasma Profile and Arginine-Stimulated GH Release

Woelfle Joachim , Schreiner Felix , Gohlke Bettina

Background: Arginine HCl infusion is commonly used in the diagnostic workup of GH deficiency. There is a paucity of data whether obesity and/or short stature modulate arginine plasma concentration and associated arginine-stimulated GH secretion following a weight-based arginine infusion protocol.Objective and hypotheses: To study whether auxiological parameters modulate the arginine plasma concentration profile and associated GH secretion in children und...

hrp0086p1-p638 | Growth P1 | ESPE2016

Retrospective Analysis of Growth Hormone (GH) Treatment Results in Children with Idiopathic Growth Hormone Deficiency (IGHD), Turner Syndrome (TS) and Small for Gestational Age (SGA) using iGRO* in a Pediatric Endocrine Practice

Partsch Carl-Joachim , Jakisch Bele , Ostendorf Anne , Stahnke Nikolaus , Wusthof Achim

Background: Quality management of GH treatment in children is important to ensure optimal treatment outcome and to save resources in the health care system. iGRO is a new internet based Medical Device to compare treatment results with predicted results according to published prediction models.Objective and hypotheses: Growth data were analyzed by iGRO for 1st and 4th year prediction in comparison to treatment results. All eligible patients of our practic...

hrp0086p1-p639 | Growth P1 | ESPE2016

Spanish ECOS Study Analysis: Socioeconomic Data, Adherence and Growth Outcomes with Case Studies

Rodriguez-Arnao Maria , Sanchez Amparo Rodriguez , Lopez Ignacio Diez , Fernandez Joaquin Ramirez , de la Vega Jose Bermudez , Ballano Virginia , Nieto Jenny Alvarez , Koledova Ekaterina

Background: The ECOS observational study in Spain (NCT01376921) aims to evaluate adherence to r-hGH therapy prescribed via the easypod™ electromechanical auto-injector device and to analyse factors that may influence adherence in paediatric patients. Easypod™ administers pre-set doses of Saizen® r-hGH and stores accurate records of each dose and injection taken, which can then be shared with the HCP for evaluation of the patient’s adherence.<...

hrp0086p1-p640 | Growth P1 | ESPE2016

Analysis of Correlation between Stem Cells (CD133+/CD45+ and CD133+/CD45) and Anthropometric Parameters of Children with Growth Hormone/Primary Insulin-Like Growth Factor 1 Deficiency

Sawicka Beata , Moniuszko Marcin , Grubczak Kamil , Singh Paulina , Radzikowska Urszula , Mikłasz Paula , Dębrowska Milena , Bossowski Artur

Introduction: Hematopoetic progenitor stem cells (HSCs, CD133+/CD45+) and very small embryonic-like stem cells (VSELs, CD133+/CD45−) can differentiate into specific immune cells. Some studies suggest that levels of HSCs and VSELs change during therapy with growth hormone (GH) or insulin-like growth factor 1 (IGF-1). GH deficiency (GHD), an endocrine disease connected with insufficient production of GH by pituitary gland, is tre...