Statin Drugs May Protect Children's Hearts
Cholesterol-Lowering Drugs May Be Safe for Use in Children at Risk
WebMD News Archive
July 20, 2004 -- Children with an inherited form of high cholesterol that leads to early heart disease may benefit from taking cholesterol-lowering statin drugs, according to new research.
The study shows two years of statin therapy significantly reduced "bad" LDL cholesterol levels and reversed some of the plaque buildup of atherosclerosis, or hardening of the arteries, in children at risk for heart disease. The cholesterol-lowering medication had no apparent negative effects on the children's growth and sexual development.
Statins are a class of drugs that block the enzyme in the liver that is responsible for making cholesterol in the body, and the cholesterol-lowering drugs are commonly used in adults with high cholesterol. But the safety and effectiveness of these drugs have not been widely studied in children.
Researchers say familial hypercholesterolemia is an inherited condition that causes LDL "bad" cholesterol levels to be abnormally high from birth onward. The disorder affects about one in 500 people and frequently leads to atherosclerosis and premature heart disease.
The findings, published in the July 21 issue of The Journal of the American Medical Association, suggest that treating children who have familial hypercholesterolemia with statin drugs may help reduce their future risk of heart disease. But more studies are needed to look at the long-term effects of statin use among children.
Statins May Help Children Lower Cholesterol Levels
The study compared the effects of treating children with this condition with the statin Pravachol or a placebo for two years. The children, who ranged in age from 8 to 18, also followed a low-fat diet and were encouraged to exercise regularly.
Researchers found that children who took the statin experienced a 24% reduction in LDL cholesterol levels compared with a 0.3% reduction among those who took the placebo.
Children who took statins also showed a trend toward regression of their atherosclerosis, while those who took the placebo showed a trend toward progression of the artery disease.
Researchers found no differences in growth, sexual maturation, and hormone levels. Serious side effects from the drugs can occur, such as muscle breakdown and liver function abnormalities. These side effects can be detected in blood tests but were not seen in the children taking statins.
"This shows the increased arterial wall thickness found in children with familial hypercholesterolemia is reversible," write researcher Albert Wiegman, MD, PhD, of the University of Amsterdam, and colleagues. "Moreover, we extensively analyzed possible adverse events and untoward influences on growth and maturation of the children and none were observed, although some of our safety outcomes may have been underpowered."
Researchers say that although this study provides "the most extensive follow-up to date, data on even longer-term safety and efficacy of statin therapy in children are needed."
In an editorial that accompanies the study, Antonio Grotto Jr., MD, DPhil, of the Weill Medical College of Cornell University in New York City, says this study shows that drug therapy may be needed in most children with familial hypercholesterolemia.
"Appropriate targeting of lifestyle and drug therapies will optimize primary prevention in this group at demonstrated risk for early coronary disease," he writes.
SOURCE: Wiegman, A. The Journal of the American Medical Association, July 21, 2004; vol 292: pp 331-337.