ESPE Yearbook of Paediatric Endocrinology

J Clin Endocrinol Metab. 2022; 107(3): 801-812. PMID: 34653252 https://pubmed.ncbi.nlm.nih.gov/34653252/Brief Summary: This clinical trial evaluated the safety and efficacy of crinecerfont, a CRF1R antagonist, in suppressing adrenal androgen secretion in adult patients with classic congenital adrenal hyperplasia (CAH) during a treatment period of 14 days.Class...

J Clin Endocrinol Metab. 2021; 106(11): e4666-e4679. PMID: 34146101https://pubmed.ncbi.nlm.nih.gov/34146101/Brief Summary: These clinical trials evaluated the safety and efficacy of tildacerfront, a CRF1R antagonist, in suppressing adrenal androgen secretion in adult patients with classical Congenital adrenal hyperplasia (CAH) during a treatment period of 12 weeks.<p class="abs...

moab@chop.edu Pediatr Blood Cancer. 2022; 69: e29674. PMID: 35338690.Brief Summary: Childhood cancer survivors (CCS), particularly those exposed to local irradiation, are at increased risk for thyroid disease and thyroid surgery. This retrospective, monocentric study compared rates of surgical complications among 42 CCS undergoing thyroid surgery compared to 596 non-CCS controls (sporadic/familial thyroid cance...

T1DI Study Group. Nat Commun. 2022 Mar 21;13(1):1514. https://pubmed.ncbi.nlm.nih.gov/35314671/Brief Summary: This study of 5 birth cohorts of individuals at high risk for type 1 diabetes (T1D) used machine learning methods to explore trajectories from autoantibodies appearance to T1D progression. They identified 11 distinct latent health states and individuals progressed according to one o...

yufang@mail.xjtu.edu.cn Front Pharmacol 2022 13:820621. doi: 10.3389/fphar.2022.820621Brief Summary: This national cross-sectional survey in Pakistan found poor availability of insulin in the public sector, even for brands manufactured by domestic companies.Access to insulin remains a major issue worldwide in low- and middl...

prahalad@stanford.edu Pediatr Diabetes 2022; 23: 228–236. doi: 10.1111/pedi.13278Brief Summary: Analysis of the multi-national SWEET diabetes registry showed that mean HbA1c in youth is highest at diagnosis and lowest at 4 to 5 months post-diagnosis. The HbA1c trajectory was lower in countries with nationalized health insurance. High gross domestic p...

N Engl J Med. 2021;385(6):503-15. doi: 10.1056/NEJMoa2107519.PubMed ID: 34170647Brief summary: this randomized control trial in 1879 adults with type 2 diabetes compared effects of once-weekly Tirzepatide (5, 10 or 15 mg) versus once-weekly Semaglutide (1 mg) on glycemic control. After 40 weeks, all doses of Tirzepatide were superior to Semaglutide on lowering HbA1c.Gluca...

Nat Hum Behav. 2022;6(1):43-54. doi: 10.1038/s41562-021-01196-4.PubMed ID: 34504299Brief summary: This study analysed a large database of emails, calendars, instant messages, video/audio calls and workweek hours routinely generated by 61 182 US Microsoft employees in the months before and after 4th March 2020, when the company mandated that all non-essential employees change to full-time w...

JAMA. 2022;327(4):350-9. doi: 10.1001/jama.2021.23686.PubMed ID: 35076666Brief summary: This study assessed the penetrance of pathogenic and loss-of-function clinical variants in 2 large population-based biobank studies. It found that the penetrance of pathogenic/loss-of-function variants was variable but generally low among such unselected populations.We are increasing...

To read the full abstract: J Clin Invest. 2020 Feb 3;130(2):641–654. doi: 10.1172/JCI127378. PMID: 31845906.Congenital pituitary hypoplasia (CPH) is a multifactorial disorder, in which the pituitary has not developed correctly. The underlying mechanisms are mostly unknown, and studies are limited by the lack of appropriate disease models. This work modelled the effect of a novel OTX2</e...