Liacouras C A, Coates P M, Gallagher P R, Cortner J A
Division of Gastroenterology and Nutrition, Children's Hospital of Philadelphia, PA 19104.
J Pediatr. 1993 Mar;122(3):477-82. doi: 10.1016/s0022-3476(05)83444-4.
We studied the effectiveness of and compliance with the use of cholestyramine in children with heterozygous familial hypercholesterolemia (FH) and familial combined hyperlipidemia (FCHL). During a 10-year period, 673 children (aged 10.5 +/- 4.0 years) were referred for evaluation of hyperlipidemia, of whom 87 (36 with FH; 51 with FCHL) were treated with cholestyramine (8 to 24 gm/day). In both groups, total cholesterol, low-density lipoprotein (LDL)-cholesterol, and apolipoprotein B levels were significantly reduced after cholestyramine use. In those with FH, plasma LDL-cholesterol levels decreased from 258 +/- 35 mg/dl (6.67 +/- 0.90 mmol/L) to 190 +/- 31 mg/dl (4.91 +/- 0.80 mmol/L); in those with FCHL, LDL-cholesterol levels dropped from 207 +/- 40 mg/dl (5.35 +/- 1.03 mmol/L) to 141 +/- 35 mg/dl (3.64 +/- 0.90 mmol/L). High-density lipoprotein-cholesterol levels were not significantly changed after cholestyramine use in either group. In the FCHL group, plasma triglyceride levels increased significantly from 81 +/- 35 mg/dl (0.92 +/- 0.40 mmol/L) to 134 +/- 42 mg/dl (1.52 +/- 0.48 mmol/L). Seven patients were lost to follow-up; 18 discontinued the medication within 1 month. Of the remaining 62 children, 59 had a good response to the drug. Of the 62 patients, 52 discontinued the medication after 21.9 +/- 10 months. Adverse effects included foul taste (73%), nausea with bloating (18%), and constipation. Cholestyramine is effective in reducing LDL-cholesterol levels in children with inherited hyperlipidemia, but the majority of children will not comply with its long-term use.
我们研究了消胆胺在杂合子家族性高胆固醇血症（FH）和家族性混合性高脂血症（FCHL）患儿中的疗效及用药依从性。在10年期间，673名儿童（年龄10.5±4.0岁）因高脂血症接受评估，其中87名（36名FH；51名FCHL）接受了消胆胺治疗（8至24克/天）。在两组中，使用消胆胺后总胆固醇、低密度脂蛋白（LDL）胆固醇和载脂蛋白B水平均显著降低。FH患儿的血浆LDL胆固醇水平从258±35毫克/分升（6.67±0.90毫摩尔/升）降至190±31毫克/分升（4.91±0.80毫摩尔/升）；FCHL患儿的LDL胆固醇水平从207±40毫克/分升（5.35±1.03毫摩尔/升）降至141±35毫克/分升（3.64±0.90毫摩尔/升）。两组使用消胆胺后高密度脂蛋白胆固醇水平均无显著变化。在FCHL组中，血浆甘油三酯水平从81±35毫克/分升（0.92±0.40毫摩尔/升）显著升高至134±42毫克/分升（1.52±0.48毫摩尔/升）。7名患者失访；18名在1个月内停药。在其余62名儿童中，59名对药物反应良好。在这62名患者中，52名在21.9±10个月后停药。不良反应包括味道难闻（73%）、恶心伴腹胀（18%）和便秘。消胆胺可有效降低遗传性高脂血症患儿的LDL胆固醇水平，但大多数儿童不会长期坚持使用。
