The recent Food and Drug Administration (FDA) approval of the first statin drug for use by children may improve treatment of high blood cholesterol for some, but it's also raising concern about inappropriate use of a powerful drug in kids.
The American Academy of Pediatrics (AAP) worries that the FDA's February approval of Merck's Mevacor (lovastatin) -- for children with a disorder that gives them very high blood cholesterol -- could fuel more statin use when there is still little long-term safety and efficacy data in children, said John Moore, a pediatric cardiologist at Philadelphia's M.C. Hahnemann Medical Center and chairman of the AAP's cardiology section. There's also no consensus on how best to treat high cholesterol in youngsters, he said. The academy is expected to write new guidelines in late July.
The most recent National Cholesterol Education Program guidelines say drug therapy should be considered in adults when LDL (or "bad") cholesterol rises above 190, or above 160 when there are two or more risk factors for cardiovascular disease. The same rules are usually followed in children, said Stephen Daniels, a pediatric cardiologist at the Cincinnati Children's Hospital Medical Center. There are no good figures on how many children have dangerous LDL levels, but the American Heart Association estimates that 10 percent of children aged 12 to 19 have high total cholesterol levels -- above 200.
Daniels predicted most pediatricians will be circumspect in prescribing Mevacor for children. "My guess is that there's not going to be an explosion in usage," he said. But others, like Moore, are less certain.
Mevacor was approved specifically for children aged 10 to 17 who have heterozygous familial hypercholesterolemia (HFH), an inherited condition that affects one in 500 Americans and puts them at very high risk for early heart disease and death. But "off-label" use of statins for uses beyond those for which they are specifically approved is common. Many pediatric cardiologists use statins already to lower cholesterol in children, and it's possible that the approval of Mevacor for children will open the door to wider pediatric use for children with milder cholesterol problems.
In Merck's two studies of boys and girls with HFH, Mevacor cut cholesterol by 25 to 30 percent -- "on par with what we see in adults," said David Orloff, director of FDA's division of metabolic and endocrine drugs. But because the trials were short-term -- boys were studied for one year, girls for six months -- it is not known if Mevacor will slow the progression of heart disease in children or have unforeseen side effects.
Pete Kwiterovich, director of the University Lipid Clinic at Johns Hopkins University Medical Center and lead investigator in both trials, said the drug showed no evidence of two common statin effects -- liver and muscle damage. Mevacor also did not appear to affect growth or sexual development during the period of study -- both particular concerns, since cholesterol plays a key role in growth and sex hormone synthesis.
Mevacor for children is the same formulation as the adult product but in a lower dose -- up to 40 milligrams a day, half that used in adults. Since the drug lost its patent protection last year, cheaper generics have become available. But the approval for use in children permits Merck to market the brand-name drug for that use, something the other statins, including the generics, may not do.
-- Alicia Ault
