For kids with inherited high cholesterol, statins may be beneficial in adulthood

Initiation of statin therapy during childhood in patients with familial hypercholesterolemia slowed the progression of carotid intima–media thickness and reduced the risk of cardiovascular disease in adulthood, according to a new study published in the New England Journal of Medicine that examined the long-term effect of statin therapy in children.

Researchers conducted a 20-year follow-up study of 214 patients with familial hypercholesterolemia, a genetic disorder characterized by high cholesterol levels, who previously participated in a placebo-controlled trial evaluating the 2-year efficacy and safety of pravastatin.

Study participants were invited for follow-up, along with their 95 unaffected siblings. Participants completed a questionnaire, provided blood samples, and underwent measurements of carotid intima–media thickness.

The incidence of cardiovascular disease among the patients with familial hypercholesterolemia was compared with that of their 156 affected parents. Of the original cohort, 86% of the patients with familial hypercholesterolemia and 81% of the siblings were seen in follow-up. The mean LDL cholesterol level in the patients had decreased from 237.3 to 160.7 mg/dL, a decrease of 32% from the baseline level. Treatment goals were achieved in 20% of the patients. The mean progression of carotid intima–media thickness over the entire follow-up period was 0.0056 mm per year in patients with familial hypercholesterolemia and 0.0057 mm per year in siblings.

In addition, the researchers found that the cumulative incidence of cardiovascular events and of death from cardiovascular causes at age 39 years was lower among the patients with familial hypercholesterolemia than among their affected parents.