ISSN 1662-4009 (online)

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Yearbook of Paediatric Endocrinology 2022

ey0019.12-11 | Hyperlipidemia | ESPEYB19

12.11. Identification and evaluation of a lipid-lowering small compound in preclinical models and in a Phase I trial

H. Wang J, Zhao J, Yan C, Xi C, Wu C, Zhao J, Li F, Ding Y, Zhang R, Qi S, Li X, Liu C, Hou W, Chen H, Wang Y, Wu D, Chen K, Jiang H, Huang H, Liu

Cell Metabolism 2022;34(5):667-80.e6. doi: 10.1016/j.cmet.2022.03.006Brief Summary: In preclinical models and a phase 1 trial, a powerful new lipid small molecule was shown to act through a mechanism distinct from those of known hypolipidemic agents. Targeting HNF-1α may be a new therapeutic strategy.Comment: Familial hypercholesterolemia (FH) is the most common...

ey0019.12-12 | Hyperlipidemia | ESPEYB19

12.12. Childhood hypertriglyceridemia: is it time for a new approach?

AP Sunil B, Ashraf

Current atherosclerosis reports 2022;24(4):265-75. doi: 10.1007/s11883-022-01000-2Brief Summary: This review article discusses emerging therapies targeted at specific genes, proteins and enzymes to selectively alter triglyceride (TG) metabolism.Comment: Hypertriglyceridemia is a long-neglected major cardiovascular disease (CVD) risk factor. Recent epidemiological dat...

ey0019.12-13 | Hyperlipidemia | ESPEYB19

12.13. Vaccine targeting ANGPTL3 ameliorates dyslipidemia and associated diseases in mouse models of obese dyslipidemia and familial hypercholesterolemia

Y Fukami H, Morinaga J, Nakagami H, Hayashi H, Okadome Y, Matsunaga E, Kadomatsu T, Horiguchi H, Sato M, Sugizaki T, Kuwabara T, Miyata K, Mukoyama M, Morishita R, Oike

Reports Medicine 2021;2(11):100446. doi: 10.1016/j.xcrm.2021.100446Brief Summary: This mouse study validated a novel, safe treatment with ANGPTL3 peptide vaccine. It was effective to improve obesity-induced dyslipidemia and fatty liver in mice, and also improved dyslipidemia and atherosclerosis in a mouse model of familial hypercholesterolemia, with no reported toxicity.<p class="abste...

ey0019.12-14 | Hyperlipidemia | ESPEYB19

12.14. Maternal hypercholesterolaemia during pregnancy affects severity of myocardial infarction in young adults

C Cacciatore F, Bruzzese G, Abete P, Russo G, Palinski W, Napoli

Eur J Prev Cardiol 2022;29(5):758-65. doi: 10.1093/eurjpc/zwab152Brief Summary: This retrospective study identified an association between high maternal cholesterol levels during pregnancy with more severe heart attacks in young adult offspring.Comment: During pregnancy, physiological increases in total cholesterol and triglyceride (TG) levels occur due to increases in i...

ey0019.12-15 | Hyperlipidemia | ESPEYB19

12.15. Awareness, diagnosis and treatment of heterozygous familial hypercholesterolemia (HeFH) Results of a US national survey

DG Block RC, Bang M, Peterson A, Wong ND, Karalis

J Clin Lipidol. 2021 Sep-Oct;15(5):682-689. PMID: 34593357 doi: 10.1016/j.jacl.2021.09.045Brief summary: This online survey of cardiologists and primary care physicians, conducted by the National Lipid Association, revealed the need to improve awareness, knowledge and treatment of heterozygous familial hypercholesterolemia (HeFH).Comment: HeFH is an autosomal dominan...

ey0019.12-16 | Hyperlipidemia | ESPEYB19

12.16. Biased Outcome reporting Guidelines for Underwhelming Studies (BOGUS) statement and checklist

GR Bauer

BMJ 2021; 375:e067350 doi: 10.1136/bmj-2021-067350Comment: Do not miss this article from the Christmas edition of The BMJ published in 2021.Short Summary: With some ethanol assistance, the generation of 13 final checklist items and a flow diagram-guided structure of the checklist was produced by a single author who had multiple opinions. These guidelines direct...