Endocrine Abstracts

Background: Treatment outcome with hydrocortisone for congenital adrenal hyperplasia (CAH) in neonates and children is highly variable. As there is no licensed formulation for children <6 years hydrocortisone capsules individually have to be compounded by local pharmacies (dose strength: 0.5–9.5 mg). The aim of this study was to characterise mass and content of these capsules in order to assess medication safety for effective and nontoxic dosing in terms of precision ...

Background: Current standard treatment for adrenal insufficiency in neonates and infants is unsatisfactory as unlicensed adult dosage formulations are used. These are generally difficult to administer and may give rise to inconsistencies in dose as the content uniformity of the dosage form cannot be verified.Methods: Infacort is a newly-developed immediate release formulation of hydrocortisone that is provided in appropriate unit dosage units (0.5, 1, 2,...

A novel solid-phase-extraction (SPE) method followed by liquid chromatography tandem mass spectrometry (LC-MS/MS) was established for the simultaneous detection and quantification of thyroid hormone profiles in one sample of human or mouse serum.Recovery during preanalytical steps was monitored by addition of an internal deuterated standard (d5-T4) to 200 μl serum, which was deproteinized by adding a precipitating agent consisting of 1% f...

Objective: CHI is the most frequent cause of severe hypoglycaemia in infants. Two distinct anatomical forms have been described which require different therapeutic strategies. We evaluated the predictive value and accuracy of integrated [18F]FDOPA-PET/CT as a new tool in identification of focal lesions in an observational study.Patients and methods: From 2005 to 2007, 73 infants and children from the UK (30) and Germany (42) with CHI were exam...

ContextPrenatal dexamethasone (Dex) therapy is used in female foetuses with congenital adrenal hyperplasia (CAH) to suppress adrenal androgen excess and prevent virilisation of the external genitalia. The prenatal dexamethasone dose of 20 µg/kg per day has been used for decades in prenatal CAH and is associated with risks for the treated mother and potentially for the unborn child. Despite the high medical need, no prospective, clinical studies had ...

Introduction: Children with adrenal insufficiency requiring hydrocortisone rely on compounded adult medication. This study aimed to evaluate the absorption, palatability and safety of Alkindi® (hydrocortisone granules in capsules for opening).Methods: The phase 3 study was an open-label, single-dose study in a total of 24 children (aged 0–6 years) with adrenal insufficiency. Fasted children were given a single dose of Alkindi&#1...

Congenital hyperinsulinism (CHI) is a cause of severe and persistent hypoglycaemia due to unregulated insulin secretion from pancreatic β-cells. Mutations in the ABCC8 and KCNJ11 genes are the most common cause of medically unresponsive CHI. Histologically there are three major subgroups, focal, diffuse and atypical. The pathophysiology of focal CHI is complex and involves a two hit process with the patient firstly inheriting a paternal ABCC8/KCNJ11 m...

Background: Data from several clinical studies have demonstrated the benefit of GH treatment in relation to bone mineral density and body composition in young adults who received GH treatment in childhood due to GHD1–3. There are recommendations to use a higher GH dose in the transition phase compared to the GH dose required during adulthood to mimic the endogenous GH secretion4,5.Methods: Data from NordiNet IOS have been analy...

Clinical Endocrinology, 2018, 88(1): 21–29 (DOI: https://doi.org/10.1111/cen.13447)...

Background: Congenital adrenal hyperplasia (CAH) is a rare condition that is associated with life long risk of adrenal crisis. Management of CAH demands a fine balance between excess glucocorticoid leading to adverse effects and too little glucocorticoid risking adrenal crises. Frequent occurrence of sick day episodes warrants dose adjustment and education regarding adrenal crisis. In a condition such as CAH it is difficult to collect sufficient data from small cohorts at a si...