A randomized, double-blind, placebo-controlled trial 1 included 214 children with familial hypercholesterolemia, aged 8 to 18 years and recruited from an academic medical referral center in the Netherlands. After initiation of a fat-restricted diet and encouragement of regular physical activity, children were randomly assigned to receive treatment with pravastatin, 20 to 40 mg/d (n = 106), or a placebo tablet (n = 108). Compared with baseline, carotid carotid intima-media thickness (IMT) showed a trend toward regression with pravastatin (mean (SD), -0.010 (0.048) mm; P =.049), whereas a trend toward progression was observed in the placebo group (mean (SD), +0.005 (0.044) mm; P =.28). The mean (SD) change in IMT compared between the 2 groups (0.014 (0.046) mm) was significant (P =.02). Pravastatin significantly reduced mean low-density lipoprotein cholesterol levels compared with placebo (-24.1% vs +0.3%, respectively; P<.001). No differences were observed for growth, muscle or liver enzymes, endocrine function parameters, Tanner staging scores, onset of menses, or testicular volume between the 2 groups.
Comment: The quality of evidence is downgraded by imprecise results (limited study size).
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