ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: Cell Metabolism 2018;27:854-868. e8People gain weight due to treatment with glucocorticoid (GC) drugs, but also in chronic stress and disrupted circadian rhythms. We now learn that fat-cell maturation increases if the trough in exposure to GC lasts less than 12 hours. A long pulse of glucocorticoids lasting 48 hours led most of the cells to differentiate under the ...

Brief summary: In this study of 699 youth 10 to < 18 years old with <2 years duration of type 2 diabetes (T2D) at 15 centers across the USA, baseline HbA1c level, and the change in HbA1c level in the first 6 months are predictors of rapid glycemic deterioration. In addition, subsequent loss of control can be predicted based on both baseline and ongoing clinical characteristics.Comment: The population of individuals with youth-onset T2D is heterog...

Brief Summary: This open-label, randomised phase 3 trial investigated the efficacy and safety of subcutaneous infusions of Dasiglucagon1, a glucagon analogue, as an add-on to standard of care (SoC) treatment in infants and children with congenital hyperinsulinism (CHI).Patients had documented CHI aged 0.6 to 10.9y, who had ≥3 episodes of hypoglycemia/week, defined as self-measured plasma glucose (SMPG) <3.9 mmol/L. Part 1 (weeks 1-4)...

Brief Summary: This study assessed the effect of growth hormone (GH) on WIP1 (wild-type p53-inducible phosphatase 1), a key mediator of the DNA damage response (DDR). GH suppressed DDR by inducing WIP1, which dephosphorylates and inactivates ATM (ataxia-telangiectasia mutated) kinase and its downstream effectors (such as CHK2, p53 and H2AX), leading to the accumulation of unrepaired DNA, which can potentially contribute to tumorigenesis.This elegant stud...

Brief Summary: This longitudinal study explored the impact of metabolic surgery on the growth hormone (GH)/IGF-1 axis and its subsequent effects on adipose tissue function. The results suggest that bariatric surgery enhances GH-IGF-1 levels, which are linked to improved adipose tissue function and a reduction in inflammation markers.This study explored how metabolic surgery influences the GH system, and how these changes are related to improvements in ad...

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...

Brief Summary: This double-blind, randomized trial, showed that 2 dosing regimens of alirocumab, a human monoclonal antibody to proprotein convertase subtilisin kexin type 9 (PCSK9), reduced LDL-C in children as young as 8 years with heterozygous familial hypercholesterolemia inadequately controlled by statins. Efficacy was sustained over 2 years, and both regimens were generally well tolerated.Comment: Heterozygous familial hypercholesterolemia (HeFH) i...

Brief summary: This French multicenter prospective study analyzed uterine volume by pelvic MRI in 88 women (age range 18–40 years), who were survivors of childhood acute leukemia treated with hematopoietic stem cell transplantation (HSCT). They were compared to 88 healthy women matched for age and parity.Conditioning regimens before HSCT included alkylating agents for 34 women and total body irradiation (TBI) for 54 women. Scans were centralized and...

Nature Metabolism. 2020;2(2):192–209. doi: 10.1038/s42255-020-0171-3.This experimental mouse study examined whether or not dedifferentiated β cells could be reversed or targeted by pharmacological intervention for diabetes remission. They identified evidence for β-cell dedifferentiation and dysfunction which could be reversed by single and combined pharmacological approach...

EMBO Rep. 2020;21:e50612. doi: 10.15252/embr.202050612.This zebrafish study identified and molecularly characterized adult transcriptionally different thyrocyte subpopulations even within the same follicle.It is well established that within a thyroid gland, follicles are heterogenous concerning functional activity. More active follicles are characterized by a high co...