Statins Found to Be Safe and Effective for the Treatment of Children With Familial Hypercholesterolemia

Statin Drug-Drug Interactions
Statin Drug-Drug Interactions
Pediatricians should be familiar with familial hypercholesterolemia and prescribe statins to treat this disorder.

Pediatricians should be familiar with familial hypercholesterolemia (FH) and prescribe statins to treat this disorder, in order to avoid associated potential long-term side effects from high cholesterol during adolescence, according to the results of a systematic review published in the Journal of Pediatric Health Care.

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 assessed 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 identified (participants’ age range, 6-18 years; sample size, 17-272 children; study length, 24 weeks-3 years; 7 studies included boys and girls).

Of 10 studies reviewed, 6 had randomized, placebo-controlled designs, all of which reported significant reductions in LDL-c concentration in participants treated with statins vs placebo (P <.001). Of those 6 studies, LDL-c goal was reached by 40% of children at 12 weeks and 60% of children at 26 weeks of statin therapy.

The remaining 4 studies had a longitudinal design and had no control group. In all, a reduction in LDL-c levels was observed, ranging from a 25% reduction associated with a year-long treatment with simvastatin (5 mg), to a 43.8% reduction associated with a 36-week treatment with atorvastatin.

Study limitations include the review of studies in which the effect of multiple statins was examined. However, as multiple statins of the same class were found to have similar results in lowering LDL-c levels, these observations indicate a high efficacy of statins in pediatric patients.

The review authors conclude that FH is a common genetic disorder which is associated with multiple comorbidities that may results in cardiovascular complications and premature death. LDL-c lowering therapies have historically been delayed until adulthood, however, a delay may cause lasting health effects.

Results from this review indicate that statins can be administered to children with FH and clinicians who care for pediatric patients should be familiar with the diagnosis and treatment of FH.


Dombalis S and Nash A. The effect of statins in children and adolescents with familial hypercholesterolemia: A systematic review. J Pediatr Health Care. 2020;S0891-5245(20)30308-4. doi:10.1016/j.pedhc.2020.11.007