Department of Medicine, Meir Medical Center, Kfar Saba, Israel.
Am J Cardiol. 2011 Jul 15;108(2):223-6. doi: 10.1016/j.amjcard.2011.03.027. Epub 2011 May 3.
Long-term data on the effects of cholesterol-lowering regimens on low-density lipoprotein cholesterol levels and cardiovascular events in patients with familial hypercholesterolemia (FH) are lacking. The present study evaluated the effectiveness of long-term intensive lipid-lowering therapy on the natural history of FH. Of approximately 1,000 adult patients with heterozygous FH treated from 1974 to 2008, the charts of 327 were randomly selected for review. FH was defined according to the Simon Broome Registry Group criteria. The recorded data included age; gender; lipid levels with diet only, with each lipid-lowering regimen, and at the most recent visit during treatment; the length of follow-up; cardiovascular events; and revascularization procedures. The lipid assay calibrations and standardization were unchanged throughout the study period. Of the 327 patients, 60% were men, the mean age at diagnosis was 38 ± 14 years, and the mean follow-up was 15 ± 8 years. The baseline and most recent low-density lipoprotein cholesterol levels during treatment were 256 ± 60 mg/dl and 116 ± 46 mg/dl, respectively, for a mean reduction of 55% from baseline (p <0.0001). At their most recent visit, 24% of all subjects were treated with statin monotherapy, 55% with a statin plus another agent, and 21% with triple therapy; 44% received a statin-ezetimibe combination. The interval between recurrent cardiovascular events tended to increase from 5.3 ± 4.8 years before treatment to 7.4 ± 6.7 years after referral (p = 0.1303). In conclusion, advances in drug therapy during the past 3 decades has led to substantial reductions in low-density lipoprotein cholesterol levels and appears to diminish the cardiovascular risk in patients with FH.
家族性高胆固醇血症（FH）患者降脂方案对低密度脂蛋白胆固醇水平和心血管事件的长期数据缺乏。本研究评估了长期强化降脂治疗对 FH 自然史的影响。在 1974 年至 2008 年期间治疗的约 1000 名杂合子 FH 成年患者中，随机选择了 327 份病历进行回顾。FH 根据 Simon Broome 登记组标准定义。记录的数据包括年龄；性别；仅饮食、每种降脂方案以及治疗期间最近一次就诊时的血脂水平；随访时间；心血管事件；以及血运重建术。整个研究期间，血脂分析校准和标准化均未改变。327 例患者中，60%为男性，诊断时的平均年龄为 38±14 岁，平均随访时间为 15±8 年。治疗期间的基线和最近的低密度脂蛋白胆固醇水平分别为 256±60mg/dl 和 116±46mg/dl，平均降低 55%（p<0.0001）。在最近一次就诊时，所有患者中有 24%接受了他汀类药物单药治疗，55%接受了他汀类药物联合另一种药物治疗，21%接受了三联疗法；44%接受了他汀类药物-依折麦布联合治疗。复发性心血管事件之间的间隔时间从治疗前的 5.3±4.8 年趋于增加到治疗后 7.4±6.7 年（p=0.1303）。总之，过去 30 年药物治疗的进步导致低密度脂蛋白胆固醇水平显著降低，似乎降低了 FH 患者的心血管风险。
