Gylling H, Siimes M A, Miettinen T A
Department of Internal Medicine, University of Helsinki, Finland.
J Lipid Res. 1995 Aug;36(8):1807-12.
In familial hypercholesterolemia (FH) the lowering of serum cholesterol levels should be started in childhood in order to prevent coronary artery disease later in life. However, treatment of children is problematic. We studied the effects of sitostanol (3 g/day) ester dissolved in rapeseed oil margarine as a hypocholesterolemic agent in one homozygous and 14 heterozygous children with FH maintained on a low cholesterol diet for 6 weeks, using a double-blind crossover design. Absorption and synthesis of cholesterol were evaluated by measuring serum plant sterol and cholesterol precursor proportions to cholesterol by gas-liquid chromatography. The compliance was good, and the children could not distinguish by taste the two margarines without and with sitostanol ester. Sitostanol margarine significantly reduced serum total, intermediate density (IDL), and low density lipoprotein (LDL) cholesterol by 11, 26, and 15%, respectively, and increased HDL/LDL cholesterol ratio by 27%. The proportions of serum delta 8-cholestenol, lathosterol, and desmosterol were significantly increased by 36, 19, and 18%, and those of serum cholestanol, campesterol, and sitosterol were significantly decreased by 9, 42 and 29%, respectively, suggesting that cholesterol absorption was decreased and synthesis was compensatorily increased. High basal precursor sterol proportions predicted a high decrease in LDL cholesterol levels. In conclusion, partial replacement of normal dietary fat consumption by sitostanol ester margarine appears to be an effective and safe hypocholesterolemic treatment in children with FH.
在家族性高胆固醇血症（FH）中，应在儿童期开始降低血清胆固醇水平，以预防日后生活中的冠状动脉疾病。然而，儿童的治疗存在问题。我们采用双盲交叉设计，研究了溶解在菜籽油人造黄油中的谷甾烷醇（3克/天）酯作为降胆固醇药物，对1名纯合子和14名杂合子FH儿童的影响，这些儿童采用低胆固醇饮食持续6周。通过气液色谱法测量血清植物甾醇和胆固醇前体与胆固醇的比例，评估胆固醇的吸收和合成。依从性良好，儿童无法通过口味区分不含和含有谷甾烷醇酯的两种人造黄油。谷甾烷醇人造黄油分别使血清总胆固醇、中密度脂蛋白（IDL）和低密度脂蛋白（LDL）胆固醇显著降低11%、26%和15%，并使高密度脂蛋白/低密度脂蛋白胆固醇比值增加27%。血清δ8-胆甾烯醇、羊毛甾醇和链甾醇的比例分别显著增加36%、19%和18%，而血清胆甾烷醇、菜油甾醇和谷甾醇的比例分别显著降低9%、42%和29%，表明胆固醇吸收减少，合成得到代偿性增加。高基础前体甾醇比例预示着低密度脂蛋白胆固醇水平的大幅降低。总之，用谷甾烷醇酯人造黄油部分替代正常饮食中的脂肪摄入，似乎是FH儿童一种有效且安全的降胆固醇治疗方法。
