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Evidence summaries

Pravastatin for Familial Hypercholesterolemia in Children

Two years of pravastatin therapy appears to induce regression of carotid atherosclerosis in children with familial hypercholesterolemia, without significant adverse effects. Level of evidence: "B"

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).

    References

    • Wiegman A, Hutten BA, de Groot E, Rodenburg J, Bakker HD, Büller HR, Sijbrands EJ, Kastelein JJ. Efficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized controlled trial. JAMA 2004 Jul 21;292(3):331-7. [PubMed]

Primary/Secondary Keywords