For 20 years, statins have been shown to be largely safe and effective, with no cumulative side effects, for adults. "What we don't know is, over decades, how safe they are for children," says Dr. Alan Lewis of Childrens Hospital Los Angeles. "We're all cautious about extrapolating data from adults to children, but that's all we have available."
For some children, statins are the only option.
Probably fewer than 1% of American children have a genetic predisposition to extremely high levels of cholesterol, a condition called hypercholesterolemia. Their childhood blood levels can soar to 500 mg/dL of total cholesterol, putting them at risk of heart attacks as early as in their 20s or 30s. These high-risk kids can develop fatty deposits in arterial walls that evolve during their teens into dangerous plaque deposits. They are prime candidates for early statin use.
And for some children -- even without a genetic predisposition to very high cholesterol -- lifestyle modifications will prove unworkable or not effective enough. Those most likely to have high cholesterol include kids with a family history of early heart disease or risk factors such as obesity, high blood pressure or diabetes.
Dr. Peter Belamarich, a pediatrician at the Children's Hospital at Montefiore in New York City, has prescribed statins to children. But he has concerns that the new guidelines will encourage more statin use even among kids who are not at highest risk. It may send a message that there is a magic pill to cure their ills.
And too much is still unknown about the long-term effect of the drugs on children. There are few studies of children on statins, and all of them short-term. Probably fewer than 1,000 kids on the drugs have been tracked, and only for six months to four years. With a study that limited, Belamarich says, "you may miss rare complications." Adult studies will never answer the question of what effect a drug has on a developing child.
One known caution for females is that statins can cause birth defects, so a woman intending to get pregnant, or a sexually active teen who may unintentionally become pregnant, should not take them.
The growing number of children who are overweight, obese, diabetic and sedentary has many public health experts worried about a looming epidemic of heart disease among young adults. "But starting medication is the last option, not the first," Lewis says. "It's what we do when all else doesn't succeed."