Should children take statin drugs to lower their cholesterol?

Most statin drugs are FDA-approved for children and teens under age 18, but only if they have a genetic condition that cause extremely high levels of LDL (bad) cholesterol. Yet in 2009, pediatricians wrote children in the U.S. at least 2.8 million prescriptions for drugs to lower cholesterol; nearly 2.3 million of them were for statins. (not including prescriptions written by family doctors, psychiatrists, and other pediatric specialists), according to an internal analysis of prescription data. After all, about one in five children and adolescents 12 to 19 years have at least one lipid abnormality, including high cholesterol, according to a recent report from the Centers for Disease Control and Prevention (CDC).

Many adults with high LDL (bad) cholesterol resign themselves to taking a statin drug when no other lifestyle changes will work—and in most cases rightly so, since the evidence is strong for adults with increased risk of heart disease or those who already have heart disease. But is the same thing true for children?

Skyrocketing obesity rates—which have more than tripled in the U.S. to 18 percent of the people between 12 and 19 in the last 30 years—have also caused higher rates of conditions normally associated with adults, including type 2 diabetes, high blood pressure and high cholesterol.

To help doctors and parents treat young people with high cholesterol, the American Academy of Pediatrics revised its guidelines two summers ago and added statins—such as: atorvastatin (Lipitor); fluvastatin (Lescol); lovastatin (Altoprev, Mevacor and generic); pravastatin (Pravachol and generic); rosuvastatin (Crestor); and simvastatin (Zocor an generic)—to the array of treatments deemed appropriate for this age group. The group also lowered its suggested minimum age for drug treatment from 10 to 8.

The findings from studies of statin use in children with this inherited condition are partly behind the hypothesis that any child with elevated LDL levels would benefit from cholesterol-lowering medication based on an analysis of the published evidence by the American Society of Hospital-Pharmacists (ASHP). Other studies include autopsy reports from children who died due to other causes and were found to have plaque buildup in their artery walls (atherosclerosis), showing how heart disease can begin at an early age. Two studies have informed this concern, according to the ASHP analysis: one known as PDAY (The Pathobiological Determinants of Atherosclerosis In Youth) and another called the Bogalusa Heart Study.

When the American Academy of Pediatrics issued its revised recommendations, it had the unenviable task of trying to balance the possible risk of cardiovascular disease and heart attacks in young adulthood—particularly as obesity rates soar in this country—with the unknown effects of drug treatments. The new recommendations might serve as a wake-up call to parents and physicians.

But when statins are prescribed for children who lack the inherited condition, their use becomes off-label. That doesn't mean it's illegal: a doctor can prescribe any drug for a purpose he or she deems appropriate.

To date, the ASHP analysis concludes that there are no long-term studies of statin use by children that establish whether or not the medications reduce the number of heart attacks or other cardiovascular events in adulthood. In general, it's worth noting that off-label use of medications in kids is very common because studies are often not done due to ethical and cost reasons.

There is also concern over the long-term potential risk for children and adolescents who use these medications for years or decades, particularly the effects on the developing central nervous system, hormone levels, immune function, and organs. Lipids play a role in brain development, and at least two statins, simvastatin (Zocor and generics) and lovastatin (Altoprev, Mevacor, and generics) can cross the blood-brain barrier and could have a direct and negative impact on such development, according to a recent editorial in the Canadian Medical Association Journal.

It appears that many of the studies that have been done on children who have inherited high cholesterol levels are short-term, whereas statin therapy, in any case, may be a lifetime drug.

Medications to lower cholesterol were the most-often-prescribed drug in the U.S. in 2009. The FDA approved them to be used with dietary modifications to reduce LDL levels ("bad") cholesterol in the bloodstream. Last year, Lipitor was the most popular branded drug in the U.S., with over 51 million prescriptions written, and the highest selling, at $7.5 billion, according to figures from IMS Health. Drugs to lower cholesterol have been a boon for pharmaceutical manufacturers, earning them more than $14 billion last year alone. Today, 22 percent of Americans 45 years and older take a cholesterol-lowering statin.

And in many cases, it's with good reason. For adults, we know that statins can help lower LDL cholesterol roughly 18 to 60 percent depending on the type chosen, individual circumstances, and dosage. And for some people, statins help to cut the risk of heart attack or death. Research has also found that they might reduce the risk of a second heart attack for people who have already had one. Statin use has been studied predominately in adult populations and, while there are indeed side effects, most are generally safe. (Read on for more detailed information in our most recent Best Buy Drug report on use of statins in adults to lower cholesterol.)

But the situation is different with children. Statins approved by the FDA for use in kids are indicated for those who have an inherited cholesterol condition (heterozygous familial hypercholesterolemia) that causes very high levels of LDL in the blood—along the lines of over 500 mg/dL, leading to artery damage and possibly heart attacks at a relatively young age.

A study published in the 2009 the medical journal Circulation estimated that the number of children who are candidates for statin drugs under the AAP guidelines is quite small. The findings showed that only 5.2 to 6.6 percent of adolescents 12 to 17 years old had elevated concentrations of LDL, and of them, only 0.8 percent (about 200,000) would qualify for cholesterol-lowering treatment.

That's good news, because making the decision to commit a child to long-term statin therapy is difficult. Consumer Reports medical advisers suggest the following considerations for parents in this situation:

For children and adolescents who have risk factors for cardiovascular disease—those who are overweight or obese (a high BMI level), have diabetes or hypertension, are smokers, or have a family history of premature heart disease, there is good reason to be concerned about a child's cholesterol level.

Improving a child's diet and increasing his or her physical activity with a goal of weight loss, if it is needed, are essential. Our advisers say that lowering a child's cholesterol level without drugs and improving their overall health is vastly preferred over medication. We suggest changing diet and exercise habits for at least two years before considering drug therapy.

For children and adolescents who don't have traditional risk factors for cardiovascular disease and are healthy otherwise but have elevated LDL levels, at this time, we would be cautious about statin use before adulthood. Total cholesterol levels naturally fluctuate throughout childhood, according to a recent analysis of children and adolescents in the U.S. published in the journal Circulation. It also found that the levels appear to peak between the ages of 8 and 10. Given that there is a substantial lack of evidence that treating high cholesterol levels would reduce the number of heart attacks among this group of low-risk children, and that there are concerns over the potential effects of statins on developing children, we don't think drug treatment is warranted.

And considering that the use of statins by adult women with elevated LDL cholesterol and no known cardiovascular disease has been questioned, one should be cautious about any recommendation that young girls with no other risks for cardiovascular disease should begin statin therapy.

Physicians are increasingly writing statin prescriptions for children. If your child is still a candidate for statin drugs after diet and lifestyle changes, parents should consider the following:

• Insist that the lowest dose is used initially.
• Monitor your child for any side effects, such as muscle pain. Statins pose a risk of a rare muscle condition called rhabdomyolysis. Symptoms include muscle pain, tenderness, soreness, or weakness, or urine that is especially dark, brown, or red in color.
• Monitor for signs of liver toxicity. Symptoms include anorexia, fatigue, jaundice, nausea, pain in upper-right-hand side of the abdomen, or vomiting.
• Make sure your child gets periodic blood tests to see if his or her LDL levels are dropping and to ensure the drug isn't causing liver damage.
• Even while taking a statin, continue healthy lifestyle changes, including modifying a child's diet, since doing so can help reduce the dosage and possibly the likelihood of side effects.

No professional organization recommends screening for all children or adolescents. But the American Academy of Pediatrics recommends screening children between 2 and 10 if they have:

• a family history of high cholesterol
• a family history of early heart disease
• a family history that is unknown
• a high body mass index (or if they're obese)
• high blood pressure
• diabetes
• or who smoke

To help parents navigate these choices, Consumer Reports medical advisers suggest the following:

• If your child is overweight or obese (according to the BMI—click here to calculate), if he or she has diabetes or high blood pressure, if there is a family history of premature heart disease, or if your child smokes cigarettes, consider having cholesterol levels measured in the course of other health-care office visits.
• If your child does not have any traditional risk factors for cardiovascular disease as stated above, and is healthy otherwise, at this time we cannot determine a compelling reason to monitor cholesterol levels during childhood. Total cholesterol levels naturally fluctuate during childhood and adolescence, and appear to peak between the ages of 8 and 10. And if a child in this group were found to have elevated cholesterol, we haven't seen evidence that treating it would result in a long-term benefit or that statin drug treatment is safe for his or her development when taken for many years.