ESPE Yearbook of Paediatric Endocrinology

Brief Summary: Circulating microRNAs (miRNA) were measured using multiplatform sequencing in longitudinal samples from 262 individuals with newly diagnosed type 1 diabetes (T1D) enrolled in the INNODIA Natural History Study. A set of miRNAs located within the T1D susceptibility chromosomal locus 14q32 distinguished 2 groups of T1D: those with higher miRNA expression showed better glycemic outcomes and immunomics profile.MicroRNAs (miRNAs) are a class of ...

Brief Summary: This retrospective, single-center study from Kobe Children’s Hospital, Japan, analyzed height SDS (main outcome) and risk of short stature (height <-2 SDS, secondary outcome) in 89 children with malignant diseases who underwent initial allogenic hematopoietic stem cell transplantation (HSCT) with different conditioning intensities. More intensive conditioning conferred a substantially higher risk of short stature at 3 years after HSCT.<p class="abst...

To read the full abstract: Nature 2017, 542, 450–455Adipose tissue is no longer considered a passive energy store. Instead, it is well established as an important endocrine contributor. Here, the authors comprehensively show that the function of the adipose tissue as an endocrine organ is not limited to classical adipokines, but also includes miRNAs and maybe other non-coding RNAs. This study...

Brief summary: This multicenter, randomized, controlled, phase 3 study compared the effects of long-acting GH (Somapacitan; 0.16 mg/kg/wk) with daily GH (Norditropin; 0.034 mg/kg/d), in GHD children. The trial was conducted over 52 weeks, followed by an ongoing 3-year single-group extension period. Similar efficacy and safety for somapacitan compared to daily GH was demonstrated over 52 weeks of treatment.Long-acting growth hormone (LAGH) formulations ha...

J Clin Endocrinol Metab. 2021 Sep 27;106(10):e3990-e4006. doi: 10.1210/clinem/dgab412. PMID: 34105732Brief Summary: This observational monocenter study analyzed cognitive scores of children with permanent (n=28, with athyreosis, ectopy or hypoplasia) vs. transient (n=11, with thyroid gland in situ) congenital hypothyroidism (CH) compared to healthy children (‘controls&#1...

To read the full abstract: J Clin Endocrinol Metab, April 2020, 105(4):e1478–e1488Silver–Russell syndrome (SRS) is a rare condition associated with pre and postnatal growth retardation. The most common causes of SRS are 11p15 ICR1 loss of methylation (LOM) and maternal uniparental disomy of chromosome 7 (mUPD7). Almost all patients with SRS have a history of intrauterine growth retardation (IUGR) and may be born small for gestational a...

To read the full abstract: PLoS Med, 2019;16:e1002744These investigators painstakingly collated individual-level data from 37 pregnancy and birth cohort studies from Europe, North America, and Australia, comprising 162,129 mothers and their children, in order to identify the optimal levels gestational weight gain in relation to risk of offspring obes...