Together with heart-healthy lifestyle habits,
statins serve as the cornerstone of primary and
secondary prevention of atherosclerotic
cardiovascular disease in adults. Several conditions, most notably
familial hypercholesterolemia (FH), cause early
dyslipidemia and
vascular disease, contributing to the development and progression of
atherosclerosis from childhood and increased cardiovascular risk. In recent decades, studies increasingly have evaluated the safety and efficacy of
statins in such high-risk youth. The strongest evidence for pediatric
statin use is for the heterozygous FH population, whereby
statin use has been shown to lower
low-density lipoprotein cholesterol effectively, slow the progression of
atherosclerosis and vascular dysfunction, and significantly reduce cardiovascular risk in early adulthood. Numerous meta-analyses and Cochrane reviews have demonstrated that attributed adverse effects, including liver toxicity,
myositis, and
rhabdomyolysis, occur no more frequently in youth receiving
statins than
placebos, with no impact on growth or development. However, further studies evaluating the long-term safety of pediatric
statin use are required. In the current review, we summarize the pediatric experience of
statin use to date, focusing on its utility for FH,
Kawasaki disease, post-
heart transplantation, and other at-risk populations. Current guidelines and indications for use are summarized, and the short- and medium-term safety experience is reviewed. Finally, a clinical approach to the indications, initiation, and monitoring of
statins in youth is provided.