ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: BMJ Open. 2019;9:e024705.This protocol paper describes an innovative phase II trial to study the effect of a single low dose of adjuvant rituximab (a chimeric anti-B-cell monoclonal antibody targeting the surface molecule CD20) compared to classical carbimazole therapy in adolescents and young adults with Graves’ disease. Carbimazole treatment will be stop...

To read the full abstract: Elife. 2018 Jun 15;7. pii: e35960.Using transgenic mice, this team discover a new role for Pituitary adenylate cyclase activating polypeptide (PACAP) in female puberty and reproduction. This neuropeptide relays nutritional state information in the hypothalamus to regulate gonadotropin-releasing hormone release.Metabolic cues play...

To read the full abstract: J Clin Endocrinol Metab. 2018; 103(8): 2998–3009.Conventional glucocorticoid (GC) therapy in adrenal insufficiency (AI) does not fully mimic the endogenous cortisol circadian rhythm, and this may adversely affect long-term health. In the recent DREAM trial (Dual Release Hydrocortisone vs. Conventional Glucocorticoid Replacement in H...

To read the full abstract: J Clin Endocrinol Metab 2018;103:1459-1469The ESPE guidelines on CH recommend an initial LT4 dose of 10-15 microgram/kg per day1. Infants with severe CH, defined by a very low pretreatment TT4 or FT4 concentration, should be treated with the highest initial dose. However, little is known about long-term effects on developmental and...

To read the full abstract: N Engl J Med 2018;378:1987-1998FGF-23 is the primary regulator of phosphate homeostasis and acts by inhibiting phosphate reabsorption in the kidney (1). Loss-of-function mutations in the gene encoding phosphate-regulating endopeptidase homolog X-linked (PHEX) results in excess circulating FGF-23, which impairs renal phosphate reabsorption causing hypophosphatemia...

To read the full abstract: Inflamm Bowel Dis. 2017;23:967-975Approximately one-third of children and adolescents with Crohn disease (CD) suffer from growth failure and delayed puberty, leading in some to psychological and social dysfunction, especially in boys. Pubertal delay in CD may also impact the normal growth spurt and lead to short adult height. The aim of therapy in children with C...

To read the full abstract: Int J Obes (Lond). 2018 Jan;42(1):102-107This cohort of n=50 adolescents who underwent bariatric surgery is highly important, since such long-term follow-up data are rare. Until today, only this study cohort described here by Ryder et al. and the Swedish nationwide study (AMOS) of adolescents with severe obesity who underwent laparoscopic Roux-en-Y gastric bypass (3) hav...

To read the full abstract: Lancet 2018;391:1174-1185Non-alcoholic fatty liver disease is highly prevalent in overweight and obese children and adults. It progresses to steatohepatitis (with raised liver transaminase levels) and fibrosis, and eventually to cirrhosis. Detecting this, excluding other causes of liver disease, and monitoring its progress t...

Brief summary: Exposure to endocrine disrupting chemicals may have adverse effects on humans. Tissue specific organoids are a helpful model to study organ specific impact of compounds alone or in combination, at different doses and during different stages of tissue development (1). While such organoid models have been developed for different tissues, such models were lacking to study differentiated thyroid follicles and thyroid tissue during development and differentiation.</p...

To read the full abstract: Am J Hum Genet. 2018 Mar 1;102(3):487-493See comment on 6.13...