ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: Diabetes Care. 2017;40:1002-1009Quality of life (QOL) measures have increasingly been considered as end-points of medical interventions. Both general QOL and health- and disease-specific quality of life can be assessed using well defined and validated questionnaires. The TEENs study is an international, cross-sectional study of youth with T1DM. 5,887 participants were follo...

To read the full abstract: Lancet Diabetes Endocrinol 2017; 5:853-63 This prospective, randomized trial investigates the benefits of iodine supplementation (200 mcg per day) in pregnant women from Thailand and India on the neurological development of their children. Iodine readily crosses the placenta and is required for the synthesis of thyroid hormones by the fetal thyroid gland. In add...

Brief summary: In this experimental study, a transdermal polymeric microneedle (MN) patch for glucose-responsive closed-loop insulin and glucagon delivery was developed. In chemically-induced type 1 diabetes (T1D) mouse and minipig models, this glucose-responsive dual-hormone MN patch demonstrated tight regulation in blood glucose.The interplay between insulin and glucagon is essential for blood glucose regulation in individuals with or without diabetes....

Brief summary: This phase 3 double-blind, parallel-group, randomized, placebo-controlled trial over 68 weeks randomised (2:1) 201 adolescents with obesity to receive semaglutide 2.4 mg once weekly or placebo. Both groups also received lifestyle intervention. The primary endpoint was the percentage change in BMI. Treatment with semaglutide produced clinically relevant reductions in BMI and body weight, and improvements in cardiovascular risk factors, which were all significantl...

Brief summary: This randomized, double-blind, phase 3 trial of youths with inadequately controlled T2D, dulaglutide treatment was superior to placebo in reducing the glycated hemoglobin level at 26 weeks.Comment: T2D in children and adolescents is an aggressive disease with early onset of complications, leading to significant morbidity and mortality. After three decades with no efficient treatment, there is finally light at the end of the tunnel.<p c...

Brief summary: This study examined glycemic control following treatment with once-weekly exenatide 2 mg (Bydureon, AstraZeneca) in youth with T2D which was not optimally controlled. At 24 weeks, exenatide was superior to placebo in lowering HbA1c (least squares mean change, −0.36% with exenatide vs. +0.49% with placebo), with a between-group difference of −0.85%.Comment: The story of the development of exenatide is fascinating. It starts with...

Nat Med. 2020 Oct;26(10):1583–1592 Abstract: https://pubmed.ncbi.nlm.nih.gov/32807933/In brief: Improved treatments for osteoarthritis and other degenerative joint diseases are urgently needed. This study demonstrates, for the first time, that the synovial microenvironment can be modified to allow resident skeletal stem cells to form hyaline articular cartilage and thereby reg...

Circulation, 2022: Jun 21;145(25):1853-1866 PMID: 35616058Brief Summary: This translational study evaluated cardiac health and lifespan in two cardiomyocyte-specific transgenic mice with either enhanced or reduced IGF-1 signaling and in human cardiac biopsies from failing and nonfailing hearts. Increased IGF1R expression was related to better cardiac performance in young mice but faster decline of cardiac function with aging. Conversely reduced ...

Cell metabolism 2022;34(2):329-45.e8. doi: 10.1016/j.cmet.2021.12.016Brief Summary: In a mouse model, time of exercise during the day was found to determine the magnitude and type of metabolic response. Different tissues responded to exercise at different times of the day.Comment: Exercise is considered a vital intervention in the prevention and treatment of individu...

N Engl J Med. 2021;385(17):1581-92. doi: 10.1056/NEJMoa2103329.PubMed ID: 34614324Brief Summary: The authors performed whole exome sequencing in 2548 children with severe obesity and identified 22 GNAS mutation carriers, almost all of which disrupted melanocortin 4 receptor (MC4R) signaling.The gene GNAS encodes the stimulatory G-protein alpha subunit pr...