Statins are effective in lowering lipids in children with heterozygous familial hypercholesterolemia (FH), but they must be used with caution, according to a Finnish team of researchers.

Vuorio and colleagues conducted a meta-analysis of 9 randomized placebo controlled studies (n=1,177 participants), with a general intervention and follow-up time of 24 weeks (ranging from 6 weeks to 2 years).

The researchers found that statins reduced the mean low-density lipoprotein (LDL) cholesterol concentration at all time points (moderate quality evidence). At all time points, serum aspartate, alanine aminotransferase, and creatinine kinase concentrations did not differ between placebo and treated groups (low quality evidence). The risks of myopathy and clinical adverse events were low and similar in both groups (low quality and moderate quality evidence, respectively).

Simvastatin improved flow-mediated dilatation of the brachial artery in 1 study (low quality evidence) and 2 years of pravastatin treatment induced significant regression in carotid intima media thickness (low quality evidence).

Diagnosis of FH is based on elevated total cholesterol and LDL cholesterol levels, or DNA-based analysis, or both. Coronary atherosclerosis has been detected in men with heterozygous FH as young as age 17 years and in women as young as 25 years. Given the premature clinical complications of atherosclerosis, the authors state that lifelong treatment is started in childhood. And although diet is the cornerstone of treatment for children with familial hypercholesterolemia, the addition of lipid-lowering medications has resulted in significant improvement in treatment. 

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The authors warned that although statin treatment for children “seems to be safe in the short term,” the long-term safety is still unknown. For this reason, “children treated with statins should be carefully monitored and followed by their pediatricians and their care transferred to an adult lipidologist once they reach 18 years of age.”

They added that large, long-term randomized controlled studies are needed so as to establish the long-term safety issues of statin treatment in children.


Vuorio A, Kuoppala J, Kovanen PT, Humphries SE, Tonstad S, Wiegman A, Drogari E, Ramaswami U. Statins for children with familial hypercholesterolemia. Cochrane Database Syst Rev. 2017 Jul 7;7:CD006401.