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Should Children Really Get Drug Therapy For Cholesterol?

By Richard N. Fogoros, M.D., About.com

Updated: July 09, 2008

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The American Academy of Pediatrics (AAP) has released new recommendations on cholesterol in children. The report, published in the July 1, 2008, issue of Pediatrics, replaces a previous policy statement issued in 1998. Since that time, childhood obesity has increased, and more children and adolescents are developing hypertension, metabolic syndrome and even type 2 diabetes (conditions that, until recently, were quite rare in nonadults).

The AAP now recommends screening for elevated lipids in children between the ages 2 and 10 who have:

  • a family history of lipid abnormalities or of premature heart disease
  • an unknown family history (adopted children)
  • other risk factors for heart disease, such as obesity, diabetes or secondhand smoke.

For children who have elevated LDL cholesterol levels or other cardiovascular risk factors, and especially for children who are obese and have elevated triglycerides and reduced HDL cholesterol levels (that is, for those who appear to have metabolic syndrome) the primary recommendation is for weight loss and exercise. These recommendations are similar to those made in 1998.

The new feature in the updated recommendations is that drug therapy (generally with statins) should be initiated in children 10 years of age or older, whose LDL levels remain elevated despite diet and exercise, as follows:

  • LDL cholesterol remains greater than 190mg/dL in children without any other risk factors
  • LDL greater than 160mg/dL in children with obesity, hypertension, around smoking, or positive family history of premature heart disease
  • LDL greater than 130mg/dL in children with diabetes

These new recommendations on treating childhood lipid abnormalities have already resulted in outpourings of skepticism and outrage. (Just have a look at the online comments from readers in this Wall Street Journal Health Blog report.)
"Recommending presumably lifelong therapy with powerful drugs in kids? What can these pediatricians be thinking? Are they nuts? Are they on the take from the drug companies?"

It is always useful to try to ignore ad hominem attacks in cases like this, where new medical recommendations are attributed to objective scientific evidence, and instead try to examine the objective evidence itself. Simply, when guidelines are based on objective data, then the suitability of those guidelines can be fully judged by an analysis of that data. On the other hand, if we are forced to judge the appropriateness of guidelines by some assessment of what motivates the authors of the guidelines, then we can never get past the subjectivity that will inevitably follow.

The basic motivation behind these new recommendations is that pediatricians are seeing more fat kids than ever before and are seeing, for the first time in children and adolescents, disorders that, until recent years, occurred almost exclusively in middle-aged adults. Such disorders include hypertension, metabolic syndrome and type 2 diabetes. Furthermore, solid evidence has now accumulated that young people with such disorders are very likely to develop premature cardiovascular disease.

Dismayed by such evidence and frustrated by largely unsuccessful attempts to manage such patients with lifestyle changes, the pediatricians have decided it's time to treat children who are developing obesity-related adult disorders more like adults. A mainstay in treating adults at high risk for cardiovascular disease is to try to correct lipid abnormalities.

The pediatricians' new recommendations mirror a recent scientific statement issued by the American Heart Association on drug therapy for lipid abnormalities in children ( available online here). That statement reviewed all the available evidence on the impact of lipid abnormalities in children and on the use of drugs to treat those abnormalities. It concluded that the time is right to expand the use of lipid-lowering drugs, especially statins, in high-risk kids. The pediatricians now seem to have followed that expert advice.

In critically reviewing the objective evidence, however, it immediately becomes clear that these new recommendations are not based on long-term, randomized and controlled outcome trials, which do not exist in children, and practically speaking, would take decades to perform (decades during which, undoubtedly, the therapies being tested will have been made obsolete by the introduction of newer therapies). Given these limitations on what is practicable, the pediatricians have taken the best evidence available (including relatively small, short-term trials of statins in children with severe lipid abnormalities, such as Familial Hypercholesterolemia, long-term outcomes and safety trials in high-risk adults and an overall assessment of all available evidence by a body of experts), and have concluded that more aggressive therapy of lipid abnormalities in children is warranted.

The bottom line? Several aspects of these new recommendations seem reasonable and supportable, but they need to be carefully interpreted in considering what's best for an individual child.

Next: So how should a parent view these new recommendations?

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