Key result
Rosuvastatin reduces LDL-C up to ~50% vs. placebo in pediatric familial hypercholesterolemia.
Why the study?
Although statins effectively reduce LDL-C and slow vascular disease progression in children with familial hypercholesterolemia, few children in prior trials achieved current LDL-C goals. This study evaluated the efficacy and safety of rosuvastatin therapy in this population.
Does rosuvastatin 5, 10, or 20 mg once daily reduce LDL-C levels safely in pubertal children ages 10 to 17 years with familial hypercholesterolemia?
RCT (n=177)
Double-blind (12-week phase), followed by 40-week open-label extension
Randomized
Does rosuvastatin 5, 10, or 20 mg once daily reduce LDL-C levels safely in pubertal children ages 10 to 17 years with familial hypercholesterolemia?
In pubertal children with familial hypercholesterolemia, rosuvastatin up to 20 mg daily is well tolerated and reduces LDL-C by up to 50%, enabling 40% of patients to reach the <110 mg/dl target.
Supports rosuvastatin in children with familial hypercholesterolemia; extends statin RCT evidence to pediatrics.
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 hypercholesterolemia. 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 apparent 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).
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Avis et al. (2010) conducted an RCT in Familial hypercholesterolemia (n=177). Rosuvastatin vs. Placebo was evaluated on Percentage reduction in LDL-C compared with placebo (38% (5 mg), 45% (10 mg), and 50% (20 mg) LDL-C reduction vs. placebo, p=<0.001). Rosuvastatin 5, 10, and 20 mg daily significantly reduced LDL-C by 38%, 45%, and 50% compared with placebo in children with familial hypercholesterolemia (p < 0.001 for each).
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