Simvastatin significantly reduces cholesterol levels in children with inherited hypercholesterolemia, according to an international study published in the October 1st rapid access issue of Circulation. The randomized, double-blind, placebo-controlled trial is the most extensive study to date of cholesterol reduction in children.

"We found that simvastatin at doses up to 40 mg is a well-tolerated and effective therapy for children with inherited high cholesterol. In addition, the drug exhibited a safety and tolerability profile similar to that seen in adults and does not influence growth and pubertal development," said lead author Saskia de Jongh, M.D.

The condition that was studied, heterozygous familial hypercholesterolemia, is common and is caused by mutations in the receptor gene for low-density lipoprotein. Affected individuals show symptoms of heart disease at a young age. If untreated, they have a 50 percent risk of developing heart disease by age 50 years.

A total of 173 affected children were included in the study. The 98 boys and 75 girls were between 9 and 18 years old and were from seven countries. They were randomized to either simvastatin or placebo. The drug was started at 10 mg daily and gradually increased to 40 mg daily.

After 48 weeks, there were significant reductions in all measures of cholesterol: low-density lipoprotein cholesterol dropped 41 percent, total cholesterol decreased 31 percent, apolipoprotein B decreased 34 percent very low-density lipoprotein declined 21 percent, and triglycerides fell 9 percent.

The drug did not appear to affect the growth or maturation of the children.

The U.S. National Cholesterol Education Program recommends cholesterol-lowering drugs for children over 10 years old whose low-density lipoprotein cholesterol remains high after dietary changes. Researchers say, however, that the long-term effectiveness of dietary changes is poor, and the lipid-lowering success of drugs is modest.

The few studies to evaluate statin therapy in children and adolescents showed good effectiveness, but "they were short-term, had a limited sample size, were mostly conducted in boys, or did not provide extensive information about growth and development," note the authors.