The safety, tolerability, and efficacy of a 12-wk treatment with
pravastatin, 5, 10, and 20 mg/d, was evaluated in 72 children with heterozygous
familial hypercholesterolemia (FH) in a double-blind, randomized and placebo-controlled study. The results show that
pravastatin was well tolerated and that adverse events were mild and equally distributed among the three treatment groups. Plasma total and
LDL cholesterol levels were significantly reduced in all
pravastatin treatment groups, in comparison with the control group; -24.6% (-28.1 to 21.0) and -32.9% (-37.0 to -28.6), for mean change and 95% confidence interval, respectively. In four children plasma
LDL cholesterol levels were reduced within normal limits for sex and age.
HDL cholesterol increased in the
pravastatin 20-mg group, +10.8% (+3.4 to +18.8), whereas plasma apo B100 and very
LDL (
VLDL) cholesterol levels were reduced within all
pravastatin-treated groups -26.8% (-31.2 [corrected] to -21.7) and -24.5% (-35.0 to -12.3). These data show that short-term
pravastatin treatment of children with FH is safe and effective, although long-term dose titration studies with
3-hydroxy-3-methylglutaryl-CoA reductase inhibitors need to be performed, to reduce plasma
LDL cholesterol levels below a predefined level. The results of these studies have to be awaited before new treatment strategies are to be considered in these children.