Efficacy and Safety of Coadministration of Ezetimibe and Simvastatin in Adolescents With Heterozygous Familial Hypercholesterolemia

Anouk van der Graaf, Cynthia Cuffie-Jackson, Maud N. Vissers, Mieke D. Trip, Claude Gagné, Genming Shi, Enrico Veltri, Hans J. Avis, John J. P. Kastelein

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121 Citations (Scopus)

Abstract

Objectives The study evaluated the efficacy and safety of long-term coadministration of ezetimibe and simvastatin in adolescents with heterozygous familial hypercholesterolemia (HeFH). Background Aggressive intervention to achieve lipid goals for adolescents with HeFH is recommended to reduce risk of premature cardiovascular disease. Methods In a multicenter, randomized, double-blind, placebo-controlled study, 248 male and female subjects ages >= 10 and <= 17 years with HeFH were randomized to receive: step 1: simvastatin 10, 20, or 40 mg/day plus ezetimibe 10 mg/day or placebo for 6 weeks, followed by step 2: simvastatin 40 mg/day plus ezetimibe 10 mg/day or placebo for 27 weeks; followed by step 3: all subjects received open-label simvastatin 10 or 20 mg/day (titrated to maximum 40 mg/day) plus ezetimibe 10 mg/day for 20 weeks. Safety was assessed throughout the study. Results Coadministered ezetimibe and simvastatin for 6 weeks (step 1) resulted in significantly greater mean reduction in low-density lipoprotein cholesterol (LDL-C) from baseline (49.5%) compared with simvastatin monotherapy (34.4%; p <0.01) in pooled dose groups and in individual dose groups (46.7% vs. 30.4%, 49.5% vs. 34.3%, 52.1% vs. 38.6%, respectively; p <0.01). At 33 weeks (step 2), ezetimibe-simvastatin subjects had a mean 54.0% reduction in LDL-C compared with a mean 38.1% reduction in simvastatin monotherapy subjects (p <0.01). At 53 weeks (step 3), the pooled reduction in LDL-C was 49.1%. All treatment regimens were well tolerated throughout 53 weeks. Conclusions Coadministration of ezetimibe with simvastatin was safe, well tolerated, and provided higher LDL-C reduction compared with simvastatin alone in adolescents with HeFH studied up to 53 weeks. (Effects of Ezetimibe With Simvastatin in the Therapy of Adolescents With Heterozygous Familial Hypercholesterolemia; NCT00129402) (J Am Coll Cardiol 2008; 52: 1421-9) (C) 2008 by the American College of Cardiology Foundation
Original languageEnglish
Pages (from-to)1421-1429
JournalJournal of the American College of Cardiology
Volume52
Issue number17
DOIs
Publication statusPublished - 2008

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