TY - JOUR
T1 - The role of microsomal triglyceride transfer protein inhibitors in the treatment of patients with familial hypercholesterolemia
T2 - Risks, benefits, and management
AU - Ahmad, Zahid
AU - Khera, Amit
PY - 2015/1/18
Y1 - 2015/1/18
N2 - Statins fail to adequately reduce low-density lipoprotein-cholesterol (LDL-C) in patients with homozygous familial hypercholesterolemia, requiring these patients to undergo weekly or bi-weekly sessions of LDL apheresis. Although efficacious, LDL apheresis is an invasive procedure with high cost and low availability, and additional options, such as inhibitors of microsomal transfer protein (MTP), may have benefit. Inhibition of MTP reduces levels of circulating cholesterol and triglycerides by preventing the formation of very-low-density lipoprotein and chylomicrons. LDL-C levels decrease by as much as 50 %. Unfortunately, adverse effects—the most common of which are gastrointestinal-related and hepatic lipid accumulation—limit broader use of the drug. Furthermore, the cardiovascular benefit of MTP inhibition remains unclear. However, MTP inhibition offers a viable additional lipid-lowering option for patients with homozygous familial hypercholesterolemia.
AB - Statins fail to adequately reduce low-density lipoprotein-cholesterol (LDL-C) in patients with homozygous familial hypercholesterolemia, requiring these patients to undergo weekly or bi-weekly sessions of LDL apheresis. Although efficacious, LDL apheresis is an invasive procedure with high cost and low availability, and additional options, such as inhibitors of microsomal transfer protein (MTP), may have benefit. Inhibition of MTP reduces levels of circulating cholesterol and triglycerides by preventing the formation of very-low-density lipoprotein and chylomicrons. LDL-C levels decrease by as much as 50 %. Unfortunately, adverse effects—the most common of which are gastrointestinal-related and hepatic lipid accumulation—limit broader use of the drug. Furthermore, the cardiovascular benefit of MTP inhibition remains unclear. However, MTP inhibition offers a viable additional lipid-lowering option for patients with homozygous familial hypercholesterolemia.
KW - Abetalipoproteinemia
KW - Familial hypercholesterolemia
KW - Homozygous familial hypercholesterolemia
KW - LDL apheresis
KW - LDL-C
KW - LDL-Receptor
KW - Lomitapide
KW - MTP
UR - http://www.scopus.com/inward/record.url?scp=84938408420&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84938408420&partnerID=8YFLogxK
U2 - 10.1007/s11883-014-0469-2
DO - 10.1007/s11883-014-0469-2
M3 - Article
C2 - 25408543
AN - SCOPUS:84938408420
SN - 1523-3804
VL - 17
JO - Current Atherosclerosis Reports
JF - Current Atherosclerosis Reports
IS - 1
M1 - 469
ER -