The effects of mipomersen, a secondgeneration antisense oligonucleotide, on atherogenic (apoB-containing) lipoproteins in the treatment of homozygous familial hypercholesterolemia

Q Medicine

Clinical Lipidology Pub Date : 2014-10-01 DOI:10.2217/clp.14.43

M. McGowan, P. Moriarty, J. Backes

引用次数: 7

Abstract

Abstract Mipomersen (Kynamro® [mipomersen sodium injection]; Genzyme, a Sanofi Company, MA, USA) is indicated as an adjunct to lipid-lowering medications and diet to reduce LDL-C, apoB, total cholesterol and non-HDL cholesterol in patients with homozygous familial hypercholesterolemia. In a 6-month Phase III trial (n = 51), mean percent change in LDL-C was -25% (95% CI: -32 to -18) versus placebo -3% (95% CI: -12 to 5; p < 0.001). The absolute mean LDL-C reduction was 113 mg/dl (2.92 mmol/l). Lipoprotein(a) was also significantly lowered by -31% (95% CI: -39 to -23) versus placebo -8% (95% CI: -19 to 3; p < 0.01). Some patients experienced transient and generally reversible hepatic effects, mild-to-moderate injection site reactions and flu-like symptoms. The mechanism of action and metabolism of mipomersen makes drug–drug interactions unlikely.

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第二代反义寡核苷酸mipomersen对纯合子家族性高胆固醇血症治疗中致动脉粥样硬化(含载脂蛋白)脂蛋白的影响

Mipomersen (Kynamro®[Mipomersen钠注射液];Genzyme, Sanofi Company, MA, USA)作为降脂药物和饮食的辅助药物，用于降低纯合子家族性高胆固醇血症患者的LDL-C、载脂蛋白ob、总胆固醇和非高密度脂蛋白胆固醇。在一项为期6个月的III期试验(n = 51)中，LDL-C的平均百分比变化为-25% (95% CI: -32至-18)，而安慰剂的平均百分比变化为-3% (95% CI: -12至5;P < 0.001)。绝对平均LDL-C降低为113 mg/dl (2.92 mmol/l)。脂蛋白(a)也显著降低了-31% (95% CI: -39至-23)，而安慰剂-8% (95% CI: -19至3;P < 0.01)。一些患者出现短暂且通常可逆的肝脏反应，轻度至中度注射部位反应和流感样症状。mipomersen的作用机制和代谢使药物-药物相互作用不太可能发生。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Clinical Lipidology 生物-生化与分子生物学

CiteScore

0.44

自引率

0.00%

发文量

审稿时长

6-12 weeks

期刊介绍： The Journal of Clinical Lipidology is published to support the diverse array of medical professionals who work to reduce the incidence of morbidity and mortality from dyslipidemia and associated disorders of lipid metabolism. The Journal''s readership encompasses a broad cross-section of the medical community, including cardiologists, endocrinologists, and primary care physicians, as well as those involved in the treatment of such disorders as diabetes, hypertension, and obesity. The Journal also addresses allied health professionals who treat the patient base described above, such as pharmacists, nurse practitioners and dietitians. Because the scope of clinical lipidology is broad, the topics addressed by the Journal are equally diverse. Typical articles explore lipidology as it is practiced in the treatment setting, recent developments in pharmacological research, reports of treatment and trials, case studies, the impact of lifestyle modification, and similar academic material of interest to the practitioner. While preference is given to material of immediate practical concern, the science that underpins lipidology is forwarded by expert contributors so that evidence-based approaches to reducing cardiovascular and coronary heart disease can be made immediately available to our readers. Sections of the Journal will address pioneering studies and the clinicians who conduct them, case studies, ethical standards and conduct, professional guidance such as ATP and NCEP, editorial commentary, letters from readers, National Lipid Association (NLA) news and upcoming event information, as well as abstracts from the NLA annual scientific sessions and the scientific forums held by its chapters, when appropriate.