Knipscheer H C, Boelen C C, Kastelein J J, van Diermen D E, Groenemeijer B E, van den Ende A, Büller H R, Bakker H D
Centre for Hemostasis, Thrombosis, Atherosclerosis and Inflammation Research, University of Amsterdam, The Netherlands.
Pediatr Res. 1996 May;39(5):867-71. doi: 10.1203/00006450-199605000-00021.
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.
在一项双盲、随机、安慰剂对照研究中，对72名杂合子家族性高胆固醇血症（FH）儿童使用普伐他汀（5、10和20毫克/天）进行12周治疗的安全性、耐受性和疗效进行了评估。结果显示，普伐他汀耐受性良好，不良事件轻微，且在三个治疗组中分布均匀。与对照组相比，所有普伐他汀治疗组的血浆总胆固醇和低密度脂蛋白胆固醇水平均显著降低；平均变化和95%置信区间分别为-24.6%（-28.1至-21.0）和-32.9%（-37.0至-28.6）。在4名儿童中，血浆低密度脂蛋白胆固醇水平降至性别和年龄对应的正常范围内。普伐他汀20毫克组的高密度脂蛋白胆固醇升高，为+10.8%（+3.4至+18.8），而在所有普伐他汀治疗组中，血浆载脂蛋白B100和极低密度脂蛋白（VLDL）胆固醇水平均降低，分别为-26.8%（-31.2[校正后]至-21.7）和-24.5%（-35.0至-12.3）。这些数据表明，对FH儿童进行短期普伐他汀治疗是安全有效的，尽管需要进行3-羟基-3-甲基戊二酰辅酶A还原酶抑制剂的长期剂量滴定研究，以将血浆低密度脂蛋白胆固醇水平降至预先定义的水平以下。在考虑对这些儿童采用新的治疗策略之前，必须等待这些研究的结果。
