Bearing in mind that it is important to think about the goal of a 50% reduction in LDL cholesterol without exposing a child to excessive medication, it is still the case that in terms of choosing medications, statins are currently the preferred treatment choice. The randomized trial data of up to 2 years duration suggest that statins are safe, and this is true for all of the important statins.
Bile acid sequestrants can be considered as supplemental treatment or if statins are not tolerated. However, poor compliance is an issue.
An intestinal cholesterol absorption inhibitor (ezetimibe) can be considered as supplemental treatment, but this should not be considered for primary therapy. This should be reserved for those in whom there is statin intolerance or the rare child with sitosterolemia where cholesterol absorption inhibitors are actually more effective than statins. Finally, niacin can be very effective but its long-term safety is unknown and because of a difficult side effect profile it is often poorly tolerated. J Clin Lipidol. 2011; 5(6).
