Mipomersen in Familial Hypercholesterolemia: An Update on Health-Related Quality of Life and Patient-Reported Outcomes

被引：33

作者：

Chambergo-Michilot, Diego ^{[1
,2
]}

Alur, Anish ^{[3
]}

Kulkarni, Saneel ^{[4
]}

Agarwala, Anandita ^{[5
]}

机构：

[1] Univ Cient Sur, Lima, Peru

[2] Torres Salud Natl Res Ctr, Dept Cardiol Res, Lima, Peru

[3] Ridge High Sch, Basking Ridge, NJ USA

[4] Harvard Univ, Harvard TH Chan Sch Publ Hlth, Boston, MA 02115 USA

[5] Baylor Scott & White Hlth Heart Hosp Baylor Plano, Cardiovasc Div, Plano, TX 75093 USA

来源：

VASCULAR HEALTH AND RISK MANAGEMENT | 2022年 / 18卷

关键词：

mipomersen; familial hypercholesterolemia; apolipoprotein B; B SYNTHESIS INHIBITOR; DENSITY-LIPOPROTEIN CHOLESTEROL; APOLIPOPROTEIN-B; LDL-CHOLESTEROL; ANTISENSE INHIBITOR; DOUBLE-BLIND; EFFICACY; SAFETY;

D O I：

10.2147/VHRM.S191965

中图分类号：

R6 [外科学];

学科分类号：

1002 ; 100210 ;

摘要：

Familial hypercholesterolemia (FH) is an autosomal dominant condition that leads to significantly elevated low-density lipoprotein cholesterol (LDL-C) levels and an elevated risk for cardiovascular disease. Mipomersen is an antisense oligonucleotide inhibitor targeted to apolipoprotein B-100 (apoB-100) mRNA that is administered via subcutaneous injection. Once administered, mipomersen causes selective degradation of the apoB-100 mRNA and inhibition of protein translation. This ultimately results in substantial reductions in LDL-C and other lipoprotein levels. Mipomersen is approved for the treatment of homozygous FH. In this review, we discuss its mechanism, current evidence, limitations of use including adverse events, and impact on health-related quality of life.

引用

页码：73 / 80

页数：8

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