SAN FRANCISCO, CA—Ezetimibe 10mg/day administered in 6–10 years old children with heterozygous familial or non-familial primary hypercholesterolemia provided significant reductions in LDL-C as well as other lipid parameters after 12 weeks of therapy, as presented at ACC.13, the American College of Cardiology’s 62nd Annual Scientific Session.
Familial hypercholesterolemia (FH) is an inherited disorder of lipoprotein metabolism and the most prevalent cause for primary dyslipidemia in children. Ezetimibe, a cholesterol absorption inhibitor, was evaluated in a multicenter, 12-week, randomized, double-blind, placebo-controlled study in 138 children aged 6–10 years diagnosed with heterozygous familial hypercholesterolemia (HeFH) or clinically significant non-familial hypercholesterolemia (non-FH LDL-C >160mg/dL). Patients were randomized 2:1 to ezetimibe 10mg (n=93) or placebo (n=45).
Primary efficacy endpoints included percent change from baseline to Week 12 in LDL-C; secondary endpoints measured other lipids and lipoproteins. Safety and tolerability were assessed in all patients who received at least one dose of the blinded drug.
Study results showed that after 12 weeks, ezetimibe significantly reduced LDL-C compared to placebo(-26.7% difference; 95% CI -30.8 to -22.7, P<0.001) and other lipids and lipoproteins. Study authors noted that the change in HDL-C was not significant. “Response in subgroups by gender, race, baseline lipids, and HeFH/non-FH was generally consistent,” reported Thomas Musliner, MD, from Merck Sharp & Dome, Whitehouse Station, NJ. No clinically meaningful differences in adverse event were observes between the two groups.
“Ezetimibe monotherapy significantly reduced LDL-C and other key lipid parameters and was generally well tolerated,” Dr. Musliner concluded. Study results show that young children who require pharmacologic intervention, but are not suitable for statin therapy, may benefit from ezetimibe monotherapy.