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Study Supports Statin Use in Children with Familial Hypercholesterolemia

CardioSmart News

Children with a genetic disorder that causes abnormally high cholesterol may benefit from early statin use, based on a recent study published in the American Heart Association journal Circulation.

Known as the CHARON (Hypercholesterolemia in Children and Adolescents Taking Rosuvastatin Open Label) study, this trial tested the safety and effectiveness of statin use in children with familial hypercholesterolemia.

Familial hypercholesterolemia (FH) is a common genetic disorder that causes abnormally high cholesterol levels of LDL or “bad” cholesterol starting at birth. This condition affects an estimated 1 in 250 people worldwide and drastically increases risk for early development of heart disease.

Fortunately, evidence suggests that cholesterol-lowering statins improve outcomes for adults with FH. However, few studies have tested the effects of early treatment in children with this genetic disorder.

As part of the CHARON study, 197 children with FH took cholesterol-lowering rosuvastatin for two years. Participants were between the ages of 6 and 18 and took varying doses of rosuvastatin, depending on their age.

They, along with a total of 65 siblings without this condition, underwent three sets of imaging tests during the study period to assess their heart’s arteries. These tests measured carotid intima-media thickness (CIMT), which measures the thickness of the inner layers of the carotid artery. Thickening of the carotid artery is a sign of heart disease, even in seemingly healthy patients.

At the start of the study, children with FH had significantly thicker carotid arteries than their unaffected siblings. However, statins appeared to significantly slow the thickening of arteries in children with FH. They worked so well, in fact, that there was no difference in arterial thickness between participants and their siblings after two years.

This is the first study of its kind to assess the effects of early statin use on CIMT in children with familial hypercholesterolemia. Based on findings, authors conclude that initiating statin treatment at an early age could have significant benefits for affected children. However, additional research is needed to see whether early statin use helps reduce future risk of heart events, which is a key goal of treatment.

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