hrp0084p3-704 | Diabetes | ESPE2015

Diabetic Ketoacidosis Treatment: Experience from a Paediatric Tertiary Centre (2004–2014)

Serra-Caetano Joana , Gata Lia , Dinis Alexandra , Cardoso Rita , Dinis Isabel , Mirante Alice

Background: Diabetic ketoacidosis (DKA) is a medical emergency. The most physiologic fluid/electrolytes replacement rates and insulin dosis are still controversial.Objective and hypotheses: To evaluate the effectiveness and security of DKA treatment. Our protocol consists of 2 h’ rehydration with 0.9% sodium chloride (NaCl), followed by insulin infusion (0.1 U/kg per h) associated to 0.45% NaCl with 5% glucose. Potassium is replaced with monophospha...

hrp0084p3-945 | GH & IGF | ESPE2015

As Great Intra as Interindividual Variability in Uptake of s.c. GH Injections in Longitudinally Followed GH Treated Children

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

Background: The variation in uptake of daily sc GH-injections is hardly known.Objective and hypotheses: There is a considerable variability in uptake of s.c. GH-injections both within and between children.Method: 65 children used (Genotropin® pen 4/16, needle 12 mm), dose 0.08–0.14 mU/kg per day within trials: TRN 87–010; 88–080; 88–177; followed yearly two–eight times 1992–1999 (n=214). ...

hrp0084fc-lb-3 | Late Breaking Abstracts | ESPE2015

Pharmacokinetic and Pharmacodynamic Studies of Topicon™ Mediated Patch Delivery of Insulin Glargine in a Streptozotocin-Induced Hairless Rat Model

Hsu Stephen , Mangleburg Carl , Yao Hua

Background: The Topicon™ patch is a needle-free novel platform technology developed to achieve truly passive transdermal delivery of insulin. Here we report pharmacodynamic (PD) and pharmacokinetic (PK) studies comparing needle injection (s.c.) vs Topicon™ mediated patch delivery of the insulin analog glargine (LANTUS®) in streptozotocin-induced hairless rats.Objective and hypotheses: We sought to develop a convenient, af...

hrp0092p1-162 | Adrenals and HPA Axis (1) | ESPE2019

Evaluation of Molecular Characteristics and Steroid Metabolomics in a Large Cohort of Children with 3β-Hydroxysteroid Dehydrogenase 2 Deficiency

Guran Tulay , Kara Cengiz , Yildiz Melek , Bitkin Eda C. , Haklar Goncagul , Lin Jen-Chieh , Gilligan Lorna C. , Barnard Lise , Keskin Mehmet , Anik Ahmet , Catli Gonul , Guven Ayla , Kirel Birgul , Tutunculer Filiz , Onal Hasan , Turan Serap , Akcay Teoman , Atay Zeynep , Baranowski Elizabeth S. , Yilmaz Gulay C. , Mamadova Jamala , Akbarzade Azad , Sirikci Onder , Aghayev AghaRza , Alkan Afra , Shackleton Cedric H.L. , Storbeck Karl H. , Baris Tugba , Arlt Wiebke , Chung Bon-Chu , Bereket Abdullah

Context: Deficiency of 3β-hydroxysteroid dehydrogenase 2 (3βHSD2) causes a very rare form of congenital adrenal hyperplasia (CAH) known as 3βHSD2 deficiency, which is a consequence of biallelic HSD3B2 gene defects. The estimated prevalence is less than 1/1,000,000 live births. Knowledge of comprehensive steroid metabolome patterns in 3βHSD2 deficiency is scarce.Objective: We aimed...

hrp0094p2-19 | Adrenals and HPA Axis | ESPE2021

Genetic analysis of paediatric primary adrenal insufficiency of unknown aetiology over 25 years

Buonocore Federica , Maharaj Avinaash , Qamar Younus , Koehler Katrin , Suntharalingham Jenifer P. , Chan Li F. , Ferraz-de-Souza Bruno , Hughes Claire R. , Lin Lin , Prasad Rathi , Allgrove Jeremy , Andrews Edward T. , Buchanan Charles R. , Cheetham Tim D. , Crowne Elizabeth C. , Davies Justin H. , Gregory John W. , Hindmarsh Peter C. , Hulse Tony , Krone Nils P. , Shah Pratik , Shaikh Mohamad G. , Roberts Catherine , Clayton Peter E. , Dattani Mehul T. , Thomas N. Simon , Huebner Angela , Clark Adrian J. , Metherell Louise A. , Achermann John C. ,

Introduction: Primary adrenal insufficiency (PAI) is a potentially life-threatening condition that requires appropriate diagnosis and treatment. The most common cause of PAI is congenital adrenal hyperplasia (CAH), but other well-established aetiologies include metabolic and autoimmune disorders, and physical damage. Mutations in more than 30 genes have now been associated with PAI, with considerable biochemical and phenotypic overlap. It is therefore importan...

hrp0095p1-328 | Growth and Syndromes | ESPE2022

A real-world study of persistence with daily growth hormone therapy among children with growth hormone deficiency in Japan

Loftus Jane , Wogen Jenifer , Benjumea Darrin , Jhingran Priti , Chen Yong , Alvir Jose , Wajnrajch Michael

Background: Paediatric growth hormone deficiency (PGHD) is treated with daily somatropin (recombinant human growth hormone) injections. High rates of discontinuation and poor adherence to treatment, which are associated with worse growth outcomes, have been documented previously, for example in the US and EU. Discontinuation of somatropin has not yet been evaluated using real-world data in Japan.Objectives: To describe d...

hrp0092p3-164 | Growth and Syndromes (to include Turner Syndrome) | ESPE2019

Longitudinal Evaluation of Audiological Pattern in Turner Syndrome

Aversa Tommaso , Bruno Rocco , Santucci Simona , Messina Maria Francesca , Borrello Simona , Scarano Emanuela , Perri Annamaria , Tamburrino Federica , Valenzise Mariella , Alibrandi Angela , Mazzanti Laura , Wasniewska Malgorzata

Objectives: To investigate prognostic markers (age, initial hearing level, karyotype, chronic hormonal therapies, and presence/absence of a mid-frequency dip influence) for hearing loss (HL) in Turner syndrome (TS).Design: Longitudinal cross-sectional and retrospective study.Study Population: 61 TS females (age range 4 - 45 yrs), diagnosed by cytogenetic analysis (49,2% monosom...

hrp0089fc9.4 | Pituitary, neuroendocrinology and Puberty 1 | ESPE2018

Neuroendocrine Morbidity after Paediatric Craniopharyngioma: a Longitudinal Single Centre Analysis of 93 Patients Over 30 Years

Dehalvi Shiraz , Guzzetti Chiara , Gan Hoong-Wei , Spoudeas Helen

Context: Craniopharyngiomas are rare, suprasellar tumours with excellent 5-year survival rates of 95%. Despite their benign histology, their tendency to invade vital nearby optic, hypothalamopituitary and vascular structures, complicates resection whilst potentially causing secondary life limiting morbidity, panhypopituitarism and premature mortality. Since 1997, conservative hypothalamic-sparing surgery and radiation to the residual tumour has replaced aggressive resection at...

hrp0089p1-p040 | Diabetes & Insulin P1 | ESPE2018

Poor Metabolic Control in Children and Adolescents with Type 1 Diabetes and Psychiatric Comorbidity

Sildorf Stine M , Breinegaard Nina , Lindkvist Emilie B , Tolstrup Janne S , Boisen Kirsten A , Teilmann Grete K , Skovgaard Anne Mette , Svensson Jannet

Objective: Type 1 diabetes in childhood is associated with an increased risk of psychiatric morbidities. We investigated predictors and diabetes outcomes in a pediatric population with psychiatric comorbidities.Research Design and Methods: Nationwide pediatric registerbased study. Data from the Danish national childhood diabetes register (DanDiabKids) and The National Patient Register were collected (1996–2015) for this population-based study. We us...

hrp0086rfc3.1 | Pituitary | ESPE2016

Endocrinopathy in Childhood Intracranial Germ Cell Tumours is Predicted by Disease Location not Treatment: 30 year Experience from a Single Tertiary Centre

Serra-Caetano Joana , Dimitrakopoulou Eftychia , Ederies Ash , Phipps Kim , Spoudeas Helen Alexandra

Background: Childhood pineal and/or suprasellar intracranial germ cell tumours (IGCT) are highly curable (>90%) with neuraxial radiation alone; international (SIOP) trials have aimed to decrease late radiation-induced neuroendocrine morbidity by substituting chemotherapy. However, without longitudinal study, disease and treatment contributions to long term outcomes remain unknown.Objective: To define tumour and treatment factors implicated in neuroen...