Abstract

Familial hypercholesterolemia (FH) is a genetic disorder that causes elevated low-density lipoprotein-cholesterol (LDL-C) levels. If undiagnosed and untreated in childhood, affected individuals can suffer premature health complications. Statins reduce the risks of complications for adults, but less is known about children. This systematic review examined the effectiveness of statin therapy for lowering LDL-C levels in children with FH. Medline Ovid, Embase, CINAHL, and Allied Health Literature Plus were searched for studies that examined the effectiveness of stains in children ages 1-18 years with heterozygous FH. Of the 706 articles identified, 10 were included in the review. Statin therapy significantly reduced LDL-C levels in pediatric patients with FH. Statins were safe and well-tolerated with minimal adverse effects. Pediatric providers should be familiar with diagnosis, treatment, and management of FH to successfully lower LDL-C levels and avoid potential long-term health effects. Evidence suggests statins are safe and effective in children with FH.

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