Long-Term Statins in Children With Familial Hypercholesterolemia

Study Questions:

What is the long-term efficacy of statins to reduce the risk of cardiovascular disease (CVD) in children with familial hypercholesterolemia (FH)?


Between 1997 and 1999, 214 children with FH were randomized in a single-center, double-blind, placebo-controlled trial to evaluate the 2-year efficacy and safety of pravastatin. All children were genetically tested; 210 (98%) had a documented pathogenic mutation in the genes encoding low-density lipoprotein (LDL) receptor or apolipoprotein B. The current study presents data on the 20-year follow-up. All 214 participants together with 95 unaffected siblings were invited to participate. Carotid intima-media thickness (IMT) was measured in addition to blood work and questionnaires. The incidence of CVD among the patients with FH was compared with that among their 156 affected parents.


Of the original cohort (n = 214), 184 participants and 77 of 95 unaffected siblings were seen in follow-up. Among the 214 patients, data on CV events including mortality were available for 203 (95%) and 214 (100%), respectively. The mean LDL cholesterol level in the patients had decreased from 237.3 to 160.7 mg/dl (from 6.13-4.16 mmol/L), which was a decrease of 32% from the baseline level. Treatment goals (LDL cholesterol <100 mg/dl [2.59 mmol/L]) were achieved in 37 patients (20%). Mean progression of carotid IMT over the entire follow-up period was 0.0056 mm per year in patients with FH and 0.0057 mm per year in siblings (mean difference adjusted for sex, −0.0001 mm per year; 95% confidence interval, −0.0010 to 0.0008). The cumulative incidence of CV events and death from CV causes at 39 years of age was lower among the patients with FH than among their affected parents (1% vs. 26% and 0% vs. 7%, respectively).


The investigators concluded that initiation of statin therapy during childhood in patients with FH slowed the progression of carotid IMT and reduced the risk of CVD in adulthood.


These data support the initiation of statin therapy during childhood for patients with FH, and suggest that lifelong lower LDL cholesterol is associated with a lower risk for CVD.

Keywords: Apolipoproteins B, Carotid Intima-Media Thickness, Cholesterol, LDL, Dyslipidemias, Hydroxymethylglutaryl-CoA Reductase Inhibitors, Hyperlipoproteinemia Type II, Mutation, Pediatrics, Pravastatin, Primary Prevention, Receptors, LDL

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