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Twenty-year follow-up of statins in children with familial hypercholesterolemia

New England Journal of Medicine Oct 24, 2019

Luirink IK, Wiegman A, Kusters DM, et al. - Given the established short-term efficacy of statin therapy in children with familial hypercholesterolemia, which is characterized by severely elevated low-density lipoprotein (LDL) cholesterol levels and premature cardiovascular disease, researchers reported a 20-year follow-up study of statin therapy in children to assess changes in the risk of cardiovascular disease in long term. Of 214 patients with familial hypercholesterolemia (genetically confirmed in 98% of the patients), who previously participated in a placebo-controlled trial evaluating the 2-year efficacy and safety of pravastatin, together with their 95 unaffected siblings, were invited for follow-up; 184 of 214 patients with familial hypercholesterolemia (86%) and 77 of 95 siblings (81%) were examined in follow-up. The analysis revealed slowing down of the progression of carotid intima-media thickness and reduction in the risk of cardiovascular disease in adulthood in relation to the initiation of statin therapy in patients with familial hypercholesterolemia was done during childhood. Overall they observed the lower cumulative incidence of cardiovascular events and of death from cardiovascular causes at 39 years of age among the patients with familial hypercholesterolemia than among their affected parents (1% vs 26% and 0% vs 7%, respectively).
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