Familial hypercholesterolemia: present and future management.
Familial hypercholesterolemia: present and future management.
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DOI：
10.1007/s11886-011-0219-9
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发表时间：
2011-12
影响因子：
3.7
通讯作者：
Hovingh, G K
中科院分区：
文献类型：
--
作者：
Sjouke, B;Kusters, D M;Kastelein, J J P;Hovingh, G K
家族性高胆固醇血症(FH)患者的特点是血浆低密度脂蛋白胆固醇(LDL-C)水平升高，并增加患早产儿心血管疾病(CVD)的风险。目前的指导方针强调需要积极降低FH患者的低密度脂蛋白-C，而他汀类药物是当前方案的基石。然而，人们热切期待更多的治疗方法，特别是对于那些不能耐受他汀类药物或达不到治疗目标的患者。在过去的几年里，我们对低密度脂蛋白代谢的了解有所提高，最近发现了越来越多的潜在的新的治疗靶点。除了新的靶点，我们还面临着新的治疗方式，如信使核糖核酸反义治疗。这些新兴疗法中的一些已被证明在降低血浆低密度脂蛋白-C水平方面有效，因此有望对心血管疾病产生有益影响。希望在不久的将来，他们将丰富我们的医疗设备，以对抗FH患者中观察到的严重血脂异常。
Patients suffering from familial hypercholesterolemia (FH) are characterized by increased plasma levels of low-density lipoprotein cholesterol (LDL-C) levels and are at increased risk for premature cardiovascular disease (CVD). Current guidelines emphasize the need to aggressively lower LDL-C in FH patients, and statins are the cornerstone in the current regimen. However, additional therapies are eagerly awaited, especially for those patients not tolerating statin therapy or not reaching the goals for therapy. Our understanding of LDL metabolism has improved over the last years and an increasing number of potential novel targets for therapy have been recently identified. Apart from novel targets, we have also been confronted with novel modalities of treatment, such as mRNA antisense therapy. Some of these emerging therapies have proven to be effective in lowering plasma LDL-C levels and are as such expected to have beneficial effects on CVD. Hopefully, they will enrich our armamentarium against the severe dyslipidemia observed in FH patients in the not too distant future.