Department of Vascular Medicine, Academic Medical Center, Amsterdam, the Netherlands.
Department of Vascular Medicine, Academic Medical Center, Amsterdam, the Netherlands.
J Am Coll Cardiol. 2016 Jul 19;68(3):252-260. doi: 10.1016/j.jacc.2016.04.054.
A statin-induced reduction of coronary artery disease (CAD) events and mortality has not been adequately quantified in patients with heterozygous familial hypercholesterolemia (FH).
This study estimated the relative risk reduction for CAD and mortality by statins in heterozygous FH patients.
The authors included all adult heterozygous FH patients, identified by the Dutch screening program for FH between 1994 and 2013, who were free of CAD at baseline. Hospital, pharmacy, and mortality records between 1995 and 2015 were linked to these patients. The primary outcome was the composite of myocardial infarction, coronary revascularization, and death from any cause. The effect of statins (time-varying) was determined using a Cox proportional hazard model, while correcting for the use of other lipid-lowering therapy, thrombocyte aggregation inhibitors, and antihypertensive and antidiabetic medication. The authors applied inverse-probability-for-treatment weighting (IPTW) to account for differences at baseline between statin users and never-users.
The authors obtained medical records of 2,447 patients, of whom 888 were excluded on the basis of age <18 years or previous CAD. Simvastatin 40 mg and atorvastatin 40 mg accounted for 23.1% and 22.8% of all prescriptions, respectively. Statin users (n = 1,041) experienced 89 CAD events and 17 deaths during 11,674 person-years of follow-up versus statin never-users (n = 518), who had 22 CAD events and 9 deaths during 4,892 person-years (combined rates 8.8 vs. 5.3 per 1,000 person-years, respectively; p < 0.001). After applying IPTW and adjusting for other medications, the hazard ratio of statin use for CAD and all-cause mortality was 0.56 (95% confidence interval: 0.33 to 0.96).
In patients with heterozygous FH, moderate- to high-intensity statin therapy lowered the risk for CAD and mortality by 44%. This is essential information in all cost-effectiveness studies of this disorder, such as when evaluating reimbursement of new lipid-lowering therapies.
在杂合子家族性高胆固醇血症（FH）患者中，他汀类药物对冠心病（CAD）事件和死亡率的降低作用尚未得到充分量化。
本研究旨在评估他汀类药物在杂合子 FH 患者中降低 CAD 和死亡率的相对风险。
研究纳入了所有在 1994 年至 2013 年期间通过荷兰 FH 筛查项目确诊、基线时无 CAD 的成年杂合子 FH 患者。1995 年至 2015 年期间的住院、药房和死亡率记录与这些患者相关联。主要结局是心肌梗死、冠状动脉血运重建和任何原因导致的死亡的复合结局。使用 Cox 比例风险模型确定他汀类药物（时变）的作用，同时校正其他降脂治疗、血小板聚集抑制剂以及降压和降糖药物的使用。研究应用逆概率治疗加权（IPTW）来校正他汀类药物使用者和未使用者之间的基线差异。
研究共获得了 2447 名患者的医疗记录，其中 888 名患者因年龄<18 岁或既往 CAD 被排除。辛伐他汀 40 mg 和阿托伐他汀 40 mg 分别占所有处方的 23.1%和 22.8%。他汀类药物使用者（n=1041）在 11674 人年的随访中发生了 89 例 CAD 事件和 17 例死亡，而他汀类药物未使用者（n=518）在 4892 人年的随访中发生了 22 例 CAD 事件和 9 例死亡（合计发生率分别为 8.8 和 5.3/1000 人年，p<0.001）。应用 IPTW 并校正其他药物后，他汀类药物治疗 CAD 和全因死亡率的风险比为 0.56（95%置信区间：0.33 至 0.96）。
在杂合子 FH 患者中，中等至高强度的他汀类药物治疗可使 CAD 和死亡率降低 44%。这对于评估新降脂治疗的报销等该疾病的所有成本效益研究至关重要。
