Rodenburg J et al. (2007) Statin treatment in children with familial hypercholesterolemia: the younger, the better. Circulation 116: 664–668

Patients with familial hypercholesterolemia have elevated LDL-cholesterol levels from birth, and atherosclerosis can develop at a young age. It is unclear, however, at what age cholesterol-lowering treatment can be initiated safely.

Rodenburg et al. conducted a follow-up study of subjects from an earlier randomized trial. Participants had taken either pravastatin or placebo for the duration of the trial (2 years), and all had been started on pravastatin at the trial's conclusion. At the end of the follow-up study, data were available for 186 children (mean age 13.7 ± 3.1 years) who had been taking pravastatin for an average of 4.5 years (range 2.1–7.4 years).

Multivariate analysis showed that the earlier statin treatment was started, the lower carotid intima media thickness (IMT)—a validated surrogate marker for atherosclerotic disease—was at follow-up. According to this model, a 0.003 mm increase in carotid IMT can be expected for every year that statin therapy is postponed. Along with duration of statin treatment, initial carotid IMT and male sex were also predictors of final IMT.

Early statin therapy seems safe as well as effective—there were no clinical or laboratory adverse events during follow-up and no obvious effects on pubertal development. Longer-term observation is required to confirm these findings and establish the optimum age for the initiation of statin therapy.