hrp0082p2-d1-286 | Bone | ESPE2014

Musculoskeletal Health in Children with Crohn’s Disease at Diagnosis: Dynamic Muscle Function, Tibia Cortical and Trabecular Bone Density and Vertebral Fracture Prevalence

Ward Leanne M , Rauch Frank , Ma Jinhui , Scharke Maya , Cosgrove Heather , Matzinger Mary Ann , Shenouda Nazih , Benchimol Eric I , Mack David R

Background: The bone mass deficit in pediatric Crohn’s disease (CD) is associated with low total body lean mass and suppression of bone turnover.Objective and hypotheses: We examined at diagnosis whether the sarcopenia is associated with leg muscle hypofunction, changes in tibia muscle–bone indices as well as overt bone strength loss (vertebral fractures, VF).Method: 70% children with CD were studied within 2 weeks of dia...

hrp0082p2-d3-309 | Bone (2) | ESPE2014

Risedronate Use in Duchenne Muscular Dystrophy: a Pilot Randomised Control Trial

Mc Sweeney Niamh , Mc Kenna Malachi , van der Kamp Susan , Kilbane Mark , Mc Donnell Ciara , Murphy Nuala , Webb David , Lynch Bryan

Background: Boys affected with Duchenne Muscular Dystrophy (DMD) have lower bone mineral density compared with unaffected boys.Objective and hypotheses: We sought to determine the effects on bone mineral density (BMD) of 1 year treatment with Risedronate and calcium/vitamin D supplementation vs calcium/vitamin D supplementation alone.Method: BMD was measured at spine and whole body. We obtained early morning fasting blood samples f...

hrp0082p2-d2-378 | Fat Metabolism & Obesity (1) | ESPE2014

Circulating Concentrations of Fibroblast Growth Factor 21 are Undetectable in Human Infants at Term Birth and Surge within Hours After Birth

Sanchez-Infantes David , Gallego-Escudero Jose Miguel , Cereijo Ruben , Diaz Marta , Aragones Gemma , Lopez-Bermejo Abel , de Zegher Francis , Villarroya Francesc , Ibanez Lourdes

Background: In rodents, fibroblast growth factor 21 (FGF21), an endocrine member of the FGF family, is mainly produced in the liver and promotes glucose oxidation in several tissues; the circulating concentrations of FGF21 rise shortly after birth. In the human, the ontogeny of circulating FGF21 is essentially unknown.Objective and hypotheses: To assess whether there is also a neonatal surge of circulating FGF21 concen...

hrp0084fc4.3 | Growth | ESPE2015

An Updated and Final Analysis of a Randomised Placebo-controlled Trial of the Effect of Oxandrolone and Timing of Pubertal Induction on Final Height in Turner Syndrome

Gault Emma-Jane , Cole Tim J , Perry Rebecca J , Casey Sarah , Paterson Wendy F , Hindmarsh Peter C , Betts Peter , Dunger David B , Donaldson Malcolm D C

Background: While GH therapy forms the mainstay of growth promoting treatment for Turner syndrome (TS), adjunctive use of oxandrolone and optimal timing of pubertal induction remain controversial. The previously published interim analysis of this randomised double-blind placebo-controlled trial demonstrated that oxandrolone and pubertal induction at 14y vs 12y significantly increased final height. However, these effects were not additive.Objective: To up...

hrp0084fc6.2 | Gonads & DSD | ESPE2015

A Role for DMRT1 in Human Primary Sex-Determination

Rojo Sandra , Murphy Mark , Lee John , Gearhart Micah , Kurahashi Kayo , Banerjee Surajit , Loeuille Guy-Andre , Zarkower David , Aihara Hideki , Bardwell Vivian , McElreavey Ken , Bashamboo Anu

Background: DMRT transcription factors are highly conserved regulators of metazoan sexual development. The role of DMRT1 in human primary sex-determination is unclear. Chromosome 9p deletions that remove one copy of DMRT1 are associated with 46,XY feminization and gonadal dysgenesis. While they suggest that DMRT1 is haploinsufficient for testicular development, these deletions usually remove other genes, including DMRT2 and DMRT3. Also, most 9p deletions cause incomplete gonad...

hrp0084p2-256 | Diabetes | ESPE2015

Exposure to Phthalates and Phenols in Relation to Gestational Blood Glucose Homeostasis

Fisher Benjamin G. , Frederiksen Hanne , Andersson Anna-Maria , Juul Anders , Thankamony Ajay , Ong Ken K. , Dunger David B. , Hughes Ieuan A. , Acerini Carlo L.

Background: Endocrine disrupting chemicals (EDCs), such as phthalates and bisphenol A (BPA), have been associated with insulin resistance (IR) and type 2 diabetes (T2D) in non-pregnant adults. By contrast, recent pilot studies of pregnant women found negative associations between phthalates and blood glucose, and a lack of association with BPA. No studies have examined gestational IR or secretion in relation to EDC exposure.Objective: To confirm these re...

hrp0084lbp-1267 | Late Breaking Posters | ESPE2015

Safety and Efficacy of Long-Acting GH (VRS-317) in Children with GHD: Effects of Dose Change in the Second Treatment Year

Bright George , Moore Wayne V , Nguyen Huong Jil , Kletter Gad B , Miller Bradley S , Fechner Patricia Y , Ng David , Humphriss Eric , Cleland Jeffrey L

Background: VRS-317, a novel fusion protein of rhGH exhibiting delayed clearance, serum half-life generally >100 h, and potential for once monthly dosing, was previously evaluated in a 6-month phase 1b/2a study of weekly, twice monthly or monthly dosing (5.0 mg/kg per month) in prepubertal GHD children (n=64).Objective and hypotheses: We evaluated whether increased VRS-317 dose from 12 to 18 months can offset the decrease in height velocitie...

hrp0094p1-33 | Fat, Metabolism and Obesity A | ESPE2021

Metabolomics in early life and the association with body composition at age 2 years

van Beijsterveldt Inge , Snowden Stuart , Myers Pernille Neve , Fluiter Kirsten de , Brix Susanne , Ong Ken , Dunger David , Hokken-Koelega Anita , Koulman Albert ,

Background and Objectives: Early life might be a critical window for adiposity programming later in life. Metabolic profile in early life may reflect this programming and correlate with later life adiposity. We investigated if metabolic profile at 3 months of age is predictive for body composition at age 2 years and if there are differences between boys and girls and between infant feeding types.Methods: In 318 healthy t...

hrp0097fc10.1 | Fetal, neonatal endocrinology and metabolism (to include hypoglycaemia) & Multisystem endocrine disorders | ESPE2023

Dasiglucagon safety in paediatric participants with CHI

Meissner Thomas , D. De León Diva , Thornton Paul , Zangen David , Mohnike Klaus , Andersen Marie , Bøge Eva , Birch Sune , Ivkovic Jelena , Banerjee Indi

Background: Congenital hyperinsulinism (CHI) is a rare disorder, which causes persistent and severe hypoglycaemia in infants and children. CHI can be treated with glucagon, but long-term use is challenging owing to its instability in aqueous solution. Dasiglucagon, a stable glucagon analogue designed for long-term use as a subcutaneous continuous infusion, is in clinical development. Here, we present dasiglucagon safety results in participants treated for up t...

hrp0097p1-343 | Pituitary, Neuroendocrinology and Puberty | ESPE2023

Facilitating Transition of Care Into Adulthood in Brain Cancer Survivors With Acquired Pediatric Growth Hormone Deficiency: Insights From an Advisory Board

Alter Craig , Boguszewski Margaret , Clemmons David , Dobri Georgiana , Geffner Mitchell , Kelepouris Nicky , Miller Bradley , Oh Rich , Shea Heidi , Yuen

Kevin

Childhood cancer survivors (CCS), particularly brain cancer survivors, are at risk of developing growth hormone deficiency (GHD) due to hypothalamic-pituitary damage from direct tumor mass effects or treatment. Optimization of testing, long-term treatment, and monitoring during care transition from pediatric to adult endocrinology providers remain challenging. A group of endocrinology experts convened to discuss these challenges, the risks and benefits of GH therapy in CCS wit...