ESPE Yearbook of Paediatric Endocrinology

Brief Summary: Data from two large registries of children with homozygous familial hypercholesterolemia (HoFH) revealed that initiating lipoprotein apheresis in childhood, compared to pharmacotherapy, improves plasma LDL-C level and reduces cardiovascular death.Comment: Homozygous familial hypercholesterolaemia (HoFH) is a rare inherited disorder, which results in extremely elevated low-density lipoprotein cholesterol (LDL-C) levels and premature atheros...

Brief summary: In May 2023, the European Atherosclerosis Society (EAS) released an update to its 2014 consensus on homozygous familial hypercholesterolemia (HoFH). The 2023 statement provides updated diagnostic criteria, screening recommendations, treatment algorithms, guidance about family planning, and new insights into the genetics of the disease.Comment: Here is a brief summary of the new findings and recommendations of the updated consensus:<p c...

Brief summary: In this double-blind, randomized, placebo-controlled trial, bempedoic acid (Nexletol) reduced the incidence of a composite cardiovascular.Comment: Bempedoic acid is an ATP citrate lyase inhibitor. ATP citrate lyase catalyses the conversion of citrate to acetyl-CoA, the fundamental substrate required for the synthesis of cholesterol and fatty acids. By inhibiting the cholesterol synthesis pathway, bempedoic acid reduces intra-hepatic LDL-C....