VRN

Objectives This study was undertaken to evaluate the efficacy and safety of rosuvastatin therapy for children with familial hypercholesterolemia. Background Familial hypercholesterolemia is a common inherited disorder causing markedly elevated low-density lipoprotein cholesterol (LDL-C) levels from birth and resulting in premature atherosclerosis. In children, statins have been shown to be effective in reducing LDL-C, restoring flow-mediated dilation, and slowing carotid intima-media thickening. However, few children in these trials achieved current LDL-C goals. Methods This study comprised a 12-week double-blind, randomized, placebo-controlled trial, followed by a 40-week open- label, titration-to-goal extension phase in 177 pubertal children, ages 10 to 17 years, with familial hypercholes- terolemia. Participants were randomly assigned to placebo or rosuvastatin 5, 10, or 20 mg once daily. Results Compared with placebo, rosuvastatin 5, 10, and 20 mg reduced LDL-C by 38%, 45%, and 50%, respectively (p 0.001 for each group vs. placebo). With a maximum allowed dose of 20 mg, 40% achieved the treatment goal of 110 mg/dl during the open-label, titration-to-goal phase. Rosuvastatin was well tolerated, with no ap- parent adverse impact on growth or development. Conclusions In children with familial hypercholesterolemia, rosuvastatin 20 mg daily reduced LDL-C by 50%. Nonetheless, only 40% attained the consensus LDL-C target of 110 mg/dl, reflecting these patients’ high baseline LDL-C levels (mean, 232 mg/dl). (Pediatric Lipid-Reduction Trial of Rosuvastatin [PLUTO]; NCT00355615) (J Am Coll Cardiol 2010;55:1121–6) © 2010 by the American College of Cardiology Foundation.