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Long-term effects of statins in children with familial hypercholesterolemia


For individuals with familial hypercholesterolemia (FH), it is uncertain whether initiating statin therapy during childhood reduces the long-term risk of developing atherosclerotic cardiovascular disease, because longitudinal data have generally been lacking. A recent report from a 20-year follow-up of children enrolled in a clinical trial evaluating early statin therapy for FH provides the first direct evidence of long-term clinical benefit in this population. Trial participants had fewer cardiovascular events by age 40 years compared with their parents who were also affected by FH (1 versus 26 percent). This finding supports our suggestion for managing FH in childhood with dietary intervention and, if cholesterol levels remain elevated despite an appropriate diet, early initiation of statin therapy.

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