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...

hrp0082p2-d1-326 | Diabetes | ESPE2014

Trends in Incidence and Prevalence of DM Type 1 in Children in Ukraine During 2002–2012

Globa Evgenia , Zelinska Nataliya

Background: The aim of the study was to describe the incidence and prevalence trends of diabetes mellitus (DM) type 1 in children of different age groups in Ukraine during 2002–2012.Objective and hypotheses: Ukraine has a 25 districts, with a population of 45 553 000, including 7 971 638 children.Method: We analyzed data based on a clinical diagnosis in child population aged 0–17 years old centrally from all regions of Uk...

hrp0082p2-d3-354 | Diabetes (2) | ESPE2014

An Infant with a Novel Kir6.2 Mutation Causing Neonatal Diabetes and Unexplained Lack of Response to Sulphonylurea

O'Connell Susan M , Mc Donald Aoife , O'Toole Norma , Bradfield Anne , Bradley Maura , Hattersley Andrew , Ellard Sian , Proks Peter , Mattis Katia K , Ashcroft Frances , O'Riordan Stephen M P

Background: Neonatal diabetes (NDM) is defined as diabetes developing before 6 months of age, affecting one in 100 000 live births. Permanent NDM is diagnosed in the first 6 months of life with no remission. The majority have a mutation in the ATP-sensitive potassium (KATP) channel (KCNJ11 in 31%, ABCC8 in 13%). Autosomal dominant and recessive forms are described. The majority of patients with NDM caused by KATP mutations respond to sulphonylureas.<p...

hrp0082p2-d1-596 | Thyroid | ESPE2014

Factors Useful to Distinguish between Children with Permanent Congenital Hypothyroidism and Transient or Permanent Hyperthyrotropinemia

Francesca Messina Maria , Ramistella Vincenzo , Aversa Tommaso , Valenzie Mariella , De Luca Filippo

Background: Screening for congenital hypothyroidism (CH) with the possibility of an early treatment has transformed the outlook for children with CH. Despite the unquestioned public health success of newborn screening programs, the management of CH is still controversial. Most patients with positive screening have permanent hypothyroidism but some of them may have transient hyperthyreotropinemia, so it is important to identify these patients in order to avoid lifelong unnecess...

hrp0082p3-d2-634 | Adrenals &amp; HP Axis (1) | ESPE2014

Prenatal Dexamethasone Use for the Prevention of Virilization in Pregnancy at Risk for Classical Congenital Adrenal Hyperplasia

Fernandez Maria Sanz , de Leon Esther Gonzalez Ruiz , Iniguez Elena Dulin , Zubicaray Begona Ezquieta , Arnao Dolores Rodriguez , Sanchez Amparo Rodriguez

Background: The most common form of congenital adrenal hyperplasia (CAH) is 21-hydroxylase deficiency, which in its severe form can cause genital ambiguity in females. This can be ameliorated by administering dexamethasone to the mother.Case: This is a family whose index case is a first son diagnosed by Madrid Newborn Screening Program of CAH with salt-wasting form due to 21-hydroxylase deficiency. Mutation analysis revealed: ‘hybrid deletion’ ...

hrp0084p2-225 | Bone | ESPE2015

Early Detection of Increased Bone Turnover among Children and Adolescents with Type 1 Diabetes Mellitus

Wahab Amina Abdel , Sharkawy Sonia El , Attia Fadia , Amin Mona

Background: Most organs including bone are affected in type 2 Diabetes (T1D) mechanisms. The exact mechanism of bone derangement is still unknown.Aim of work: i) Assessment of Pyridinoline crosslinks as a bone resorption marker and alkaline phosphatase as a bone formation marker in T1D in children & adolescents. ii) To determine the effect of glycemic control and disease duration on bone turnover.Subjects and methods: 39 T1D pa...

hrp0084p2-564 | Thyroid | ESPE2015

Are Children with Congenital Primary Hypothyroidism Overtreated?

Sechter Claire , Vasseur Francis , Cartigny-Maciejewski Maryse , Gueorguieva Iva , Stuckens Chantal , Weill Jacques

Background: Treatment of congenital primary hypothyroidism (CPHT) fluctuates between two opposite risks for the neuro-cognitive development in a critical window during the neonatal period, under- and over-treatment.Objective: To investigate the relevance of current international recommendations of 10–15 μg/kg per day for L-thyroxine (lT4) doses in CPHT.Method: fT4 and TSH were measured...

hrp0084p3-686 | Diabetes | ESPE2015

A Rare Reason of Hyperinsulinism: Münchausen Syndrome by Proxy

Yesilkaya Ediz , Akin Onur , Sari Erkan , Macit Enes , Akar Cagdas , Gun Husamettin

Background: Drug intoxication is one of the rare reasons of hyperinsulinism; it may occur not only accidentally but also deliberately.Objective and hypotheses: Herein, we report a case who presented with factitious hyperinsulinaemic hypoglycemia and diagnosed with Münchausen syndrome by proxy (MSBP) which is an uncommon condition of child abuse.Method: A 7-year-old girl was referred to our department due to hyperglycemic and h...

hrp0084p3-713 | Diabetes | ESPE2015

Sirolimus Therapy in an Infant with Persistent Hyperinsulinaemic Hypoglycaemia

Lim Yvonne Yijuan , Sng Andrew Anjian , Hussain Khalid , Loke Kah-Yin

Background: Persistent hyperinsulinaemic hypoglycaemia (HH) of infancy (PHHI) is a rare condition which presents with severe hypoglycaemia during the neonatal period. For medically unresponsive forms of diffuse HH, subtotal pancreatectomy was previously the only treatment option, with the potential consequences of recurrence of hyperinsulinism, diabetes mellitus and exocrine pancreatic insufficiency. The novel use of sirolimus treatment was recently reported in four infants wh...

hrp0084p3-883 | Fat | ESPE2015

Investigating Predisposing Factors for Childhood Obesity

Magana Maria , Routsi Kleopatra , Zyga Sofia , Panoutsopoulos George , Ioannidis Anastasios , Tsironi Maria , Pistikou Anna-Maria , Kougioumtzi-Dimoliani Dafni-Eleni , Kolovos Petros , Gil Andrea Paola Rojas

Background: Childhood obesity is considered to be an epidemic in developed countries that can negatively affect children’s health and psychology.Aims and objectives: To investigate the nutritional and environmental factors that lead to the presence of childhood obesity and its complications.Methods: A total of 949 students, 3–12 years old, living in Sparta–Greece, have participated in our research. Their lifestyle an...