The Rationale, Indications, Safety, and Use of Statins in the Pediatric Population

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.