In a recent article in the Journal of Pediatric Health Care, Dombalis et al conducted a systematic review to explore the effectiveness of statin therapy for lowering LDL-C levels in children with familial hypercholesterolemia (FH). Publication databases were searched through 2020 for articles in which the effect of statin therapy on lowering levels of low-density lipoprotein cholesterol (LDL-C) were reviewed in pediatric patients with FH. 

A total of 10 studies conducted in the United States, the Netherlands, Finland, Canada, Hungary, Austria, Japan, and South Africa were recognized (participants’ age range was between 6 and 18 years; sample size, 17 to 272 pediatric patients; study length, 24 weeks to 3 years; seven studies included boys and girls). 

Of the 10 studies reviewed, six had randomized, placebo-controlled designs, all of which reported substantial decreases in LDL-C concentration in participants treated with statins versus placebo (P <.001). Of those six studies, LDL-C goal was achieved by 40% of children at 12 weeks and 60% of children at 26 weeks of statin therapy. The other four studies had a longitudinal design and had no control group. In all, a decline in LDL-C levels was noted, ranging from a 25% decrease linked with a year-long treatment with simvastatin (5 mg) to a 43.8% reduction linked with a 36-week treatment with atorvastatin. 

The authors noted that their review illustrated that FH is a common genetic disorder associated with multiple comorbidities that may cause cardiovascular complications and premature death. They also noted that LDL-C–lowering therapies have traditionally been postponed until adulthood; however, a delay may cause lasting health effects.  

The researchers concluded that pediatric providers should be acquainted with diagnosis, treatment, and management of FH to efficaciously lower LDL-C levels and avert potential long-term health effects, and the clinical data suggests statins are safe and effective in pediatric patients with FH.

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