TY - JOUR
T1 - Mipomersen in Familial Hypercholesterolemia
T2 - An Update on Health-Related Quality of Life and Patient-Reported Outcomes
AU - Chambergo-Michilot, Diego
AU - Alur, Anish
AU - Kulkarni, Saneel
AU - Agarwala, Anandita
N1 - Publisher Copyright:
© 2022 Chambergo-Michilot et al.
PY - 2022
Y1 - 2022
N2 - 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.
AB - 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.
KW - Apolipoprotein B
KW - Familial hypercholesterolemia
KW - Mipomersen
UR - http://www.scopus.com/inward/record.url?scp=85125136270&partnerID=8YFLogxK
U2 - 10.2147/VHRM.S191965
DO - 10.2147/VHRM.S191965
M3 - Artículo
C2 - 35221690
AN - SCOPUS:85125136270
SN - 1176-6344
VL - 18
SP - 73
EP - 80
JO - Vascular Health and Risk Management
JF - Vascular Health and Risk Management
ER -
