ESPE Abstracts

Background: The incidence of non-alcoholic fatty liver disease (NAFLD) is dramatically increasing among children worldwide. The gold standard for diagnosis is the liver biopsy. Reliable serum markers are lacking. Recently, circulating miRNAs have been studied as biomarkers of disease progression. Specifically, miR-122 was proposed as predictive marker for liver disease in adults, while no data are available for children.Objective and hypotheses: To inves...

Objective: Age and height at treatment start, target height, GH dose, and first year treatment response are among known criteria of GH deficiency (GHD) good responders (final adult height [FAH] >−2 standard deviation score [S.D.S.]) to GH treatment (GHT). The authors investigated whether the same criteria are applicable to SGA patients based on real-life ongoing French registry data.Methods: 291 SGA children treated with Norditro...

Background: Animal studies have reported that exposure to synthetic glucocorticoids (sGCs) may lead to inter- and transgenerational effects on offspring phenotype. However, many of these studies are poorly designed, do not account for possible non-epigenetic confounds, and cannot determine the mechanism(s) by which gamete epimutations are induced.Objectives: Firstly, to determine whether developmental exposure of mice to...

Background: Type 1 diabetes mellitus (T1DM) continues to pose serious health risks with devastating long-term complications. UK management and control of T1DM in children and young people (CYP) remains amongst the poorest in Europe and significant variations in diabetes health outcomes are evident. In 2012–13 a Best Practice Tariff (BPT) for paediatric diabetes care was introduced and a National Peer Review Quality Assurance programme (DQuINS) developed. Both were develop...

Background: Endocrine disruptors (ED) can alter endocrine function. ED have become a part of everyday life and are found among phytoestrogens, active ingredients in pharmaceuticals, and additives or contaminants in food, personal care products, cosmetics, plastics and textiles.Objective: To describe cases of children with signs of early puberty who are exposed to ED.Method: Observational study of patients affected of premature pube...

Background: Bisphenol A (BPA), a ubiquitous endocrine-disrupting chemical, has been shown to exert transgenerational effects on adiposity, metabolism, and reproductive outcomes in rodents, indicating epigenetic inheritance. Recent studies of other environmental insults (stress and high-fat diet) have demonstrated a mechanistic role for spermatozoal microRNAs in the transgenerational transmission of acquired paternal traits.Objective and hypotheses: To as...

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. These models confirm that IGF-1 (and IGF-1 SDS) varies during the dosing interval. One critical clinical and research issue is when to optimally ...

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...

Introduction: Increasing incidence of male reproductive disorders may be due to fetal exposure to putative endocrine disruptor chemicals (EDCs), such as phthalates and phenols. Anogenital Distance (AGD) is a biomarker of fetal androgen action in animals, and has recently been linked to testicular dysgenesis syndrome in humans.Objective: To examine the relationship between prenatal phthalate and phenol exposure and birth AGD in male infants.<p class="...

Background, objective, and hypotheses: OPKO Biologics has produced a long-acting human GH (hGH), MOD-4023, containing copies of a naturally-occurring C-terminal peptide (CTP) to markedly increase GH’s in vivo residence. We describe the construction and validation of a pharmacokinetic (PK)/pharmacodynamics (PD) model to characterise the relationship between MOD-4023 dose, MOD-4023 serum concentrations (Cserum), and IGF1 responses in healthy adults, GH-deficient (G...