Statin Drug Helps Kids Who Inherit High Cholesterol

It reduces arterial wall thickness and blood fat levels

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HealthDay Reporter

TUESDAY, July 20, 2004 (HealthDayNews) -- For some unlucky people, high cholesterol is passed from one generation to the next; children who inherit the disorder, known as familial hypercholesterolemia, are at great risk of having a heart attack as early as their mid-20s.

"They develop coronary artery disease at a relatively young age," explained Dr. Albert Wiegman, a pediatric cardiologist at the Academic Medical Center in Amsterdam, the Netherlands.

But the class of cholesterol-lowering drugs known as statins holds promise for these kids.

A two-year study finds children who took the drug pravastatin had significant improvement in the thickness of the walls of their carotid arteries, which supply blood to the brain. They also had sharply reduced levels of low-density lipoprotein (LDL) cholesterol, the "bad" kind that clogs vessels.

What's more, the drug worked without any apparent adverse effects on the children's growth, sexual maturation, hormone levels or liver or muscle tissue -- at least over the period of the study.

"It seems to be safe and it works," said Wiegman, lead author of the study, which appears in the July 21 issue of the Journal of the American Medical Association. "It makes the vessel walls thinner, and perhaps they [will] have a normal life expectancy."

In an editorial in the same journal, cardiologist and statin expert Dr. Antonio M. Gotto Jr., dean of the Weill Medical College of Cornell University in New York City, concluded the benefit of treating high-risk youngsters with statins exceeds the potential risk.

"In the case of familial hypercholesterolemia," he wrote, "the promise of reducing future cardiovascular morbidity and mortality, as well as future demands on acute care and more expensive medical approaches, would make aggressive treatment of high-risk youth patients a worthwhile long-term initiative."

Under normal conditions, people inherit two genes responsible for making LDL receptors -- the "fishing rods," as Wiegman describes them -- that remove cholesterol from the blood.

But in people with familial hypercholesterolemia, an abnormal gene is passed along. As a result, half of their LDL receptors are defective or missing, explains MEDPED (Make Early Diagnosis to Prevent Early Death), a Salt Lake City-based nonprofit that works to help families with inherited high cholesterol disorders. The condition results in abnormally elevated levels of LDL cholesterol beginning at birth.

An estimated 10 million people worldwide suffer from the condition, resulting in premature heart disease and death if not diagnosed and treated properly, says MEDPED.

"You have to prevent this disorder as early as possible, in my opinion," Wiegman said.

The study involved 214 children, aged 8 to 18, who were recruited between December 1997 and October 1999 and followed for two years. After encouraging every participant to exercise and eat a fat-restricted diet, investigators randomly put half of the children on 20 milligrams or 40 milligrams of pravastatin, depending on the child's age; the other half received a placebo.

Before-and-after ultrasound images were taken to measure the change in the thickness of the walls of patients' carotid arteries -- a marker for the fatty plaque build-up that leads to atherosclerosis. After two years, kids on the placebo had increased arterial wall thickness, while children on pravastatin showed significant regression.

LDL cholesterol levels dropped 24.1 percent in the statin group but rose 0.3 percent in the placebo group.

"That's promising because the LDL cholesterol-lowering, we could expect, but it's important that it does something to the vessel walls," Wiegman noted.

Although the study is believed to be the most extensive yet, longer studies are needed to demonstrate the long-term safety and efficacy of statin therapy in children, the authors said.

More information

Visit MEDPED, at the University of Utah, to learn more about inherited cholesterol disorders.

SOURCES: Albert Wiegman, M.D., Ph.D., pediatric cardiologist, Academic Medical Center, University of Amsterdam, the Netherlands; Make Early Diagnosis to Prevent Early Death (MEDPED), Salt Lake City, Utah; July 21, 2004, Journal of the American Medical Association

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