ESPE Yearbook of Paediatric Endocrinology

J Clin Endocrinol Metab. 2020; 105(9): 3103–3107.https://pubmed.ncbi.nlm.nih.gov/32629476/In this narrative review, the authors discuss the potential role of novel regenerative therapies for the treatment of adrenal insufficiency, including gene therapy and cell replacement strategies. More specifically, the authors discuss the heterogeneity in adrenal function in patients with Addison...

Diabetes Care. 2020;43(11):e172–e173. doi: 10.2337/dc20-1633During the first phase of the COVID-19 pandemic, the DPV (the German diabetes registry database) study group asked whether or not the COVID-19 lockdown had affected the incidence of pediatric T1D in Germany (1). More than 210 German pediatric diabetes centers contributed their data on pediatric patients with T1D with an onset at ag...

Brief summary: Using data from the Type 1 Diabetes Intelligence (T1DI) cohort (n=24 662), this prospective study aimed to identify optimal ages for initial islet autoantibody (IAb) screening to predict the development of clinical type 1 diabetes (T1D). The identified optimal screening ages were 2 years and 6 years, with sensitivity of 82% and positive predictive value of 79% for T1D by age 15 years.Screening for T1D is a growing research topic a...

JAMA. 2020;323(4):339–351. doi: 10.1001/jama.2019.21565This study aimed to determine the prevalence of pre-symptomatic T1D in young children participating in a public health screening program for islet autoantibodies and the risk for progression to clinical T1D in children carrying multiple autoantibodies. On screening, 0.03% were found to have clinical T1D. Of the 0.31% who were foun...

NIDDK Type 1 Diabetes TrialNet Study Group.Diabetes. 2022 Apr 1;71(4):610-623. https://pubmed.ncbi.nlm.nih.gov/35316839/Brief Summary: This is a comprehensive review on general population screening, a current hot topic in the field of type 1 diabetes (T1D). The authors provide a critical overview of the rationale for population screening, arguments for and against it, current efforts to gui...

Nature. 2021;600(7890):720-6. doi: 10.1038/s41586-021-04137-3.PubMed ID: 34880500Brief summary: This mouse study identifies FABP4 (‘Fabkin’) as a new fat-derived hormone that regulates insulin-producing beta cells in the pancreas.We have learnt over the recent years that body fat is not simply a passive energy storage tissue, but instead secretes a number o...

To read the full abstract: JAMA. 2020 Jan 28;323(4):339–351. doi: 10.1001/jama.2019.21565. PMID: 31990315It is unclear how many children in the general population have features of anti-islet cell autoimmunity without later developing type 1 diabetes (T1DM). This public health screening program determined the population prevalence of islet cell autoantibodies (ICA) and the risk for pro...

To read the full abstract: Pediatr Diabetes. 2018;19:1471–1480It has been debated whether or not new technologies, in particular the combination of insulin pumps and continuous glucose testing, will improve patient satisfaction and quality of life rather than only metabolic control and risk of microvascular complications. Indeed, the use of continuous subcutaneous insulin infusion (...

To read the full abstract: Cell Rep. 2018; 22(5): 1236-1249Primary or secondary adrenal insufficiency (AI) results from adrenal failure or impairment of the hypothalamic-pituitary axis, respectively. The most frequent cause of primary AI is autosomal recessive congenital adrenal hyperplasia (CAH). Patients with AI need life-long treatment with exogenous steroids, which can be challenging, ...

Brief summary: In this longitudinal study, blood glucose trajectories and their relationship with autoantibodies appearance were assessed in 1050 children with high genetic risk of type 1 diabetes (T1D) between 4 months and 3.6 years. Post-prandial blood glucose levels increased around 2 months prior to autoantibody seroconversion, with further increases thereafter, suggesting that islet autoimmunity co-occurs or follows insults on the β-cells.This ...