hrp0094fc9.6 | Growth Hormone and IGFs | ESPE2021

Impact of pre-transplant serum-IGF-1 on hematopoietic stem cell transplantation outcome in pediatric cancer patients

Betzmann Deborah , Doring Michaela , Blumenstock Gunnar , Erdmann Friederike , Grabow Desiree , Lang Peter , Binder Gerhard ,

Background: Hematopoietic stem cell transplantation (HSCT) is associated with severe medical complications and variable outcome depending on the recipient’s disease stage and health condition. As serum-IGF 1 is decreased in patients with severe illness, after major surgery, in malnutrition, and other conditions that cause catabolic metabolism, it may serve as predictive biomarker for transplant outcome.Individuals and Methods: Based...

hrp0094p1-40 | Fat, Metabolism and Obesity A | ESPE2021

Liraglutide for the Management of Childhood Obesity

Apperley Louise , Erlandson-Parry Karen , Gait Lucy , Laing Peter , Senniappan Senthil ,

Introduction: The prevalence of childhood obesity is continuing to increase worldwide and has become a major health concern. There are a number of serious complications to childhood obesity with significant long-term health implications if not managed in a timely matter. These complications include type 2 diabetes mellitus (T2DM), obstructive sleep apnoea (OSA), idiopathic intracranial hypertension (IIH) and non-alcoholic fatty liver disease (NAFLD). At presen...

hrp0094p1-88 | Pituitary A | ESPE2021

Pituitary microadenomas in childhood – is follow-up with diagnostic imaging necessary?

Borghammar Camilla , Tamaddon Askan , Erfurth Eva-Marie , Sundgren Pic C , Siesjo Peter , Nilsson Margareta , Elfving Maria ,

Aim: We aimed to investigate the growth potential of pituitary microadenomas and cystic lesions < 10mm in children, and to evaluate how reproducible the measurements were on magnetic resonance imaging (MRI).Design: Retrospective observational study.Methods: 74 children were included < 18 years at first pituitary MRI, and diagnosed with a microadenoma (microadenoma producing ACTH, GH or TSH ...

hrp0094p2-50 | Adrenals and HPA Axis | ESPE2021

First case in Lebanon of homozygous mutation in CYP 11B2 leading to isolated aldosterone deficiency.

Azar Ahlam , El-Rahi Hiba , Makhoul Kevin , Makhoul Peter , Megarbane Andre ,

Isolated hypoaldosteronism is a rare cause of salt wasting in infancy and may be life-threatening, especially in the newborn. Isolated aldosterone deficiency results from loss of activity of aldosterone synthase encoded by CYP 11B2 gene. We reported the case of a newborn, who presented at the age of 4 weeks with severe dehydration, vomiting and hypotonia. Blood investigations showed severe hyponatremia, hyperkaliemia and metabolic acidosis. The familial history revealed a firs...

hrp0094p2-263 | Growth hormone and IGFs | ESPE2021

Pituitary gland volume measured by Magnetic Resonance Imaging as diagnostic predictor of persistent Childhood-onset growth hormone deficiency

Corredor-Andres Beatriz , Walton-Betancourth Sandra , Brain Caroline , Butler Gary , Hindmarsh Peter , Dattani Mehul ,

Introduction: Pituitary imaging is important for the evaluation of hypothalamo-pituitary axis defects in patients diagnosed with childhood-onset growth hormone deficiency (CO-GHD). Published evidence shows that there is a close relationship between structural changes in the pituitary gland and growth hormone deficiency.Aim: To evaluate the relationship between clinical, laboratory and magnetic resonance imaging of the pi...

hrp0094p2-283 | Growth and syndromes (to include Turner syndrome) | ESPE2021

Assessment of genetic defects, baseline characteristics and adverse events reported in the Increlex® registry

Polak Michel , Bang Peter , Perrot Valerie , Sert Caroline , Storr Helen L. , Woelfle Joachim ,

Background: Certain genetic defects in the growth hormone (GH)/insulin-like growth factor 1 (IGF-1) axis are associated with severe primary IGF-1 deficiency (SPIGFD) and short stature. Detection of genetic defects may confirm short stature aetiology alongside clinical/biochemical features. Increlex® is a recombinant human IGF-1 (rhIGF-1) approved for children/adolescents with SPIGFD.Methods: The Increlex<...

hrp0097fc9.3 | Diabetes and insulin 2 | ESPE2023

A novel case of hypoglycaemia secondary to a pro-insulin processing disorder

Amin Nadia , Wark Gwen , Church David , Burling Keith , Barker Peter , Williams Gemma , O'Rahilly Stephen

Background: Hypoglycaemia in patients with a high concentration of insulin and low concentration of C-peptide can be secondary to administration of exogenous insulin. This often results in safeguarding measures, with significant consequences for the patient and family. We report a novel case of a patient with symptomatic hypoglycaemia. Initial results suggested high insulin and low C-peptide levels, but subsequent testing revealed a pro-insulin processing diso...

hrp0097p1-278 | Fetal, Neonatal Endocrinology and Metabolism | ESPE2023

Exploration of O-link protein biomarkers in children born after IUGR and early impaired developmental changes in heart function

Änghagen Olov , Rudholm Feldreich Tobias , Ärnlöv Johan , Bang Peter

We recently assessed systolic heart function in children from IUGR and normal control pregnancies and reported early developmental impairment of left ventricular longitudinal strain – a sensitive echocardiographic measure – during the first 3 months of life in IUGR children. In accordance with previous studies, this suggest that the increased cardiovascular risk later in life imposed by IUGR/SGA may, at least to some extent, be primary and not entirely secondary to...

hrp0097p1-110 | Growth and Syndromes | ESPE2023

Comparison of long-term height outcomes in pediatric patients with growth hormone deficiency receiving once weekly somatrogon with those of matched patients treated with once-daily somatropin in the Kabi/Pfizer International Growth Study (KIGS)

Paliwal Yuvika , Carlsson Martin , Zhang Richard , Lee Peter , Korth-Bradley Joan , Taylor Carrie , Cara Jose

Objectives: Somatrogon is a long-acting recombinant human growth hormone (GH) approved in the EU and other countries for once-weekly treatment of pediatric patients with GH deficiency (GHD). In this analysis, height outcomes of somatrogon-treated patients in a phase 3 trial (CP-4-006) were compared with historical data from matched somatropin-treated patients enrolled in KIGS.Methods: In trial CP-4-006, patients were ran...

hrp0098s12.3 | The impact of gonadotrophin dysregulation on neurocognition | ESPE2024

Studying the genetic interplay between GnRH deficiency and associated neurocognitive disorders by combining in silico, in vitro and in vivo tools.

Oleari Roberto , Lettieri Antonella , Amoruso Federica , Scheiffele Peter , Howard Sasha , Ruhrberg Christiana , Cariboni Anna

Gonadotropin releasing hormone (GnRH) neurons embryonically originate in nasal placode and migrate to hypothalamus, where they release GnRH to control reproduction. Defective GnRH neuron development or function leads to GnRH deficiency (GD), which is often associated to neurodevelopmental and neurological syndromes, including epilepsy, ataxia, intellectual disabilities and deafness. In addition, a register-based study showed correlation between delayed puberty and neurodevelop...