20-Year Follow-up of Statins in Children with Familial Hypercholesterolemia

作者：Ilse K. Luirink, Albert Wiegman, D. Meeike Kusters, Michel H. P. Hof, Jaap W. Groothoff, Eric de Groot, John J.P. Kastelein, Barbara A. Hutten · 发表于：New England Journal of Medicine · 年份：2019 · DOI：10.1056/nejmoa1816454 · 被引用次数：659 · 研究领域：Lipoproteins and Cardiovascular Health、Cholesterol and Lipid Metabolism、Lipid metabolism and disorders

BACKGROUND: Familial hypercholesterolemia is characterized by severely elevated low-density lipoprotein (LDL) cholesterol levels and premature cardiovascular disease. The short-term efficacy of statin therapy in children is well established, but longer follow-up studies evaluating changes in the risk of cardiovascular disease are scarce. METHODS: We report a 20-year follow-up study of statin therapy in children. A total of 214 patients with familial hypercholesterolemia (genetically confirmed in 98% of the patients), who were previously participants in a placebo-controlled trial evaluating the 2-year efficacy and safety of pravastatin, were invited for follow-up, together with their 95 unaffected siblings. Participants completed a questionnaire, provided blood samples, and underwent measurements of carotid intima-media thickness. The incidence of cardiovascular disease among the patients with familial hypercholesterolemia was compared with that among their 156 affected parents. RESULTS: Of the original cohort, 184 of 214 patients with familial hypercholesterolemia (86%) and 77 of 95 siblings (81%) were seen in follow-up; among the 214 patients, data on cardiovascular events and on death from cardiovascular causes were available for 203 (95%) and 214 (100%), respectively. The mean LDL cholesterol level in the patients had decreased from 237.3 to 160.7 mg per deciliter (from 6.13 to 4.16 mmol per liter) - a decrease of 32% from the baseline level; treatment goals (LDL cholester...