SAFETY AND EFFICACY OF TREATMENT OF PEDIATRIC CHOLESTERYL ESTER STORAGE DISEASE WITH LOVASTATIN
SAFETY AND EFFICACY OF TREATMENT OF PEDIATRIC CHOLESTERYL ESTER STORAGE DISEASE WITH LOVASTATIN
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DOI：
10.1203/00006450-199211000-00012
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发表时间：
1992-11-01
影响因子：
3.6
通讯作者：
SPEIRS, J
中科院分区：
文献类型：
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作者：
GLUECK, CJ;LICHTENSTEIN, P;SPEIRS, J
本研究的目的是前瞻性评估洛伐他汀治疗治疗开始时年龄为 11.6 岁和 5 岁的兄弟姐妹的胆固醇酯贮积病的安全性和有效性。 男性先证者的平均总胆固醇和 LDL 胆固醇分别为 7.4 和 5.68 mmol/L，仅通过饮食，洛伐他汀 40 mg/d 在 3.3 年内分别下降 30% 至 5.2（p 小于或等于 0.001）和 31% 至 3.9 mmol/L（小于或等于 0.001），同时得到缓解肝脾肿大。他的妹妹服用洛伐他汀 20 毫克/天，持续 1.5 年，总胆固醇和低密度脂蛋白胆固醇有所下降，但并不显着；治疗后她的肝脾肿大也减轻了。洛伐他汀的耐受性良好，没有明显的副作用或并发症，并且肝功能测试或肌酸磷酸激酶没有不良变化。在治疗期间，两个孩子的身高和体重均出现正常增长和预期增长。洛伐他汀似乎是治疗小儿胆固醇酯贮积病的安全有效的方法。
Tha aim of this study was to prospectively assess the safety and efficacy of lovastatin in the treatment of cholesteryl ester storage disease in siblings who were ages 11.6 and 5 y at the beginning of treatment. Mean total and LDL cholesterol in the male proband, 7.4- and 5.68 mmol/L, respectively, on diet alone, fell 30% to 5.2 (p less-than-or-equal-to 0.001) and 31% to 3.9 mmol/L (less-than-or-equal-to 0.001) on lovastatin 40 mg/d over 3.3 y, with simultaneous resolution of hepatosplenomegaly. In his sister, on lovastatin 20 mg/d for 1.5 y, total and LDL cholesterol fell, but not significantly; her hepatosplenomegaly was also reduced on treatment. Lovastatin was well tolerated without overt side effects or complications and without adverse changes in liver function tests or creatine phosphokinase. Normal and expected accretion of height and weight occurred during the treatment period for both children. Lovastatin appears to be a safe and effective treatment for pediatric cholesteryl ester storage disease.