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Statin Therapy Is Effective and Safe for Children with Hypercholesterolemia

Medication and lifestyle modification can brighten the outlook for adult health in affected children.

Statins are commonly used to treat adults with hypercholesterolemia, but the safety and efficacy of these drugs for children are not well studied. Investigators placed 214 children (mean age, 13 years) with familial hypercholesterolemia (LDL-C 155 mg/dL and demonstration of the LDL-receptor-gene mutation) on a regimen of dietary fat restriction and physical activity. The children were then randomized to receive placebo or pravastatin (10 or 20 mg/day) for 2 years.

At study end, declines in total and LDL cholesterol levels were significantly greater among pravastatin recipients than among placebo recipients. Echosonographic examination of carotid intima-media thickness showed that treated children had a trend toward regressed thickness, whereas children receiving placebo had a trend toward progressed thickness. Children in both groups had similar safety profiles: There were no differences in intellectual, pubertal, or physical development at the end of the study. Results of endocrine hormone studies and liver function studies were also similar in treated and untreated children.

Comment: Results of this elegant, well-controlled study show that statins are effective and well tolerated in children with genetic hypercholesterolemia. The findings also show that a low-fat diet, exercise, and statin therapy can benefit the carotid vascular wall during childhood. If, as the poet says, "the child is father to the man," this news suggests an improved health outlook in adulthood for these patients.

— F. Bruder Stapleton, MD

Published in Journal Watch Pediatrics and Adolescent Medicine August 30, 2004

Citation(s):

Wiegman A et al. Efficacy and safety of statin therapy in children with familial hypercholesterolemia: A randomized controlled trial. JAMA 2004 Jul 21; 292:331-7.

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