Vascular Health and Risk Management (Feb 2022)
Mipomersen in Familial Hypercholesterolemia: An Update on Health-Related Quality of Life and Patient-Reported Outcomes
Abstract
Diego Chambergo-Michilot,1,2 Anish Alur,3 Saneel Kulkarni,4 Anandita Agarwala5 1Universidad Científica del Sur, Lima, Peru; 2Department of Cardiology Research, Torres de Salud National Research Center, Lima, Peru; 3Ridge High School, Basking Ridge, NJ, USA; 4Harvard T.H. Chan School of Public Health, Harvard University, Boston, MA, USA; 5Cardiovascular Division, Baylor Scott and White Health Heart Hospital Baylor Plano, Plano, TX, USACorrespondence: Anandita Agarwala, Division of Cardiology, Center for Cardiovascular Disease Prevention, Baylor Scott & White Heart Hospital Baylor Plano, 1100 Allied Dr, Plano, TX, 75093, USA, Tel +1 469 814 3278, Email [email protected]: 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.Keywords: mipomersen, familial hypercholesterolemia, apolipoprotein B
