메뉴 건너뛰기




Volumn 7, Issue , 2012, Pages 29-38

Mipomersen: Evidence-based review of its potential in the treatment of homozygous and severe heterozygous familial hypercholesterolemia

Author keywords

Antisense oligonucleotide; Apolipoprotein B; Statin intolerance

Indexed keywords

AMINOTRANSFERASE; ANACETRAPIB; ANTILIPEMIC AGENT; ANTISENSE OLIGONUCLEOTIDE; APOLIPOPROTEIN B; BILE ACID SEQUESTRANT; CHOLESTERYLESTER TRANSFER PROTEIN INHIBITOR; DALCETRAPIB; EVACETRAPIB; EZETIMIBE; HIGH DENSITY LIPOPROTEIN CHOLESTEROL; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE INHIBITOR; LOW DENSITY LIPOPROTEIN CHOLESTEROL; LOW DENSITY LIPOPROTEIN RECEPTOR; MICROSOMAL TRIGLYCERIDE TRANSFER PROTEIN INHIBITOR; MIPOMERSEN; SIMVASTATIN; TORCETRAPIB; TRIACYLGLYCEROL; UNCLASSIFIED DRUG;

EID: 84870409612     PISSN: 15551741     EISSN: None     Source Type: Journal    
DOI: 10.2147/CE.S25239     Document Type: Review
Times cited : (32)

References (41)
  • 1
    • 33947679772 scopus 로고    scopus 로고
    • Mechanisms of disease: Genetic causes of familial hypercholesterolemia
    • DOI 10.1038/ncpcardio0836, PII NCPCARDIO0836
    • Soutar AK, Naoumova RP. Mechanisms of disease: genetic causes of familial hypercholesterolemia. Nat Clin Pract Cardiovasc Med. 2007;4(4):214-225. (Pubitemid 46502094)
    • (2007) Nature Clinical Practice Cardiovascular Medicine , vol.4 , Issue.4 , pp. 214-225
    • Soutar, A.K.1    Naoumova, R.P.2
  • 2
    • 0013321009 scopus 로고
    • Familial hypercholesterolemia: Defective binding of lipoproteins to cultured fibroblasts associated with impaired regulation of 3-hydroxy-3- methylglutaryl coenzyme A reductase activity
    • Brown MS, Goldstein JL. Familial hypercholesterolemia: defective binding of lipoproteins to cultured fibroblasts associated with impaired regulation of 3-hydroxy-3-methylglutaryl coenzyme A reductase activity. Proc Natl Acad Sci U S A. 1974;71(3):788-792.
    • (1974) Proc Natl Acad Sci U S A. , vol.71 , Issue.3 , pp. 788-792
    • Brown, M.S.1    Goldstein, J.L.2
  • 3
    • 0015890890 scopus 로고
    • Familial hypercholesterolemia: Identification of a defect in the regulation of 3-hydroxy-3-methylglutaryl coenzyme A reductase activity associated with overproduction of cholesterol
    • Goldstein JL, Brown MS. Familial hypercholesterolemia: identification of a defect in the regulation of 3-hydroxy-3-methylglutaryl coenzyme A reductase activity associated with overproduction of cholesterol. Proc Natl Acad Sci U S A. 1973;70(10):2804-2808.
    • (1973) Proc Natl Acad Sci U S A. , vol.70 , Issue.10 , pp. 2804-2808
    • Goldstein, J.L.1    Brown, M.S.2
  • 4
    • 0029090626 scopus 로고
    • Independent mutations at codon 3500 of the apolipoprotein B gene are associated with hyperlipidemia
    • Gaffney D, Reid JM, Cameron IM, et al. Independent mutations at codon 3500 of the apolipoprotein B gene are associated with hyperlipidemia. Arterioscler Thromb Vasc Biol. 1995;15(8):1025-1029.
    • (1995) Arterioscler Thromb Vasc Biol , vol.15 , Issue.8 , pp. 1025-1029
    • Gaffney, D.1    Reid, J.M.2    Cameron, I.M.3
  • 7
    • 33645103550 scopus 로고    scopus 로고
    • Sequence variations in PCSK9, low LDL, and protection against coronary heart disease
    • Cohen JC, Boerwinkle E, Mosley TH Jr, Hobbs HH. Sequence variations in PCSK9, low LDL, and protection against coronary heart disease. N Engl J Med. 2006;354(12):1264-1272.
    • (2006) N Engl J Med. , vol.354 , Issue.12 , pp. 1264-1272
    • Cohen, J.C.1    Boerwinkle, E.2    Mosley Jr., T.H.3    Hobbs, H.H.4
  • 8
    • 79956288028 scopus 로고    scopus 로고
    • Treatment of adults with familial hypercholesterolemia and evidence for treatment: Recommendations from the National Lipid Association Expert Panel on Familial Hypercholesterolemia
    • Robinson JG, Goldberg AC. Treatment of adults with familial hypercholesterolemia and evidence for treatment: recommendations from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Clin Lipidol. 2011;5(Suppl 3):S18-S29.
    • (2011) J Clin Lipidol , vol.5 , Issue.SUPPL. 3
    • Robinson, J.G.1    Goldberg, A.C.2
  • 10
    • 78449281377 scopus 로고    scopus 로고
    • Efficacy and safety of more intensive lowering of LDL cholesterol: A meta-analysis of data from 170,000 participants in 26 randomised trials
    • Baigent C, Blackwell L, Emberson J, et al. Efficacy and safety of more intensive lowering of LDL cholesterol: a meta-analysis of data from 170,000 participants in 26 randomised trials. Lancet. 2010;376(9753):1670-1681.
    • (2010) Lancet , vol.376 , Issue.9753 , pp. 1670-1681
    • Baigent, C.1    Blackwell, L.2    Emberson, J.3
  • 11
    • 26244432388 scopus 로고    scopus 로고
    • Efficacy and safety of cholesterol-lowering treatment: Prospective meta-analysis of data from 90,056 participants in 14 randomised trials of statins
    • Baigent C, Keech A, Kearney PM, et al. Efficacy and safety of cholesterol-lowering treatment: prospective meta-analysis of data from 90,056 participants in 14 randomised trials of statins. Lancet. 2005;366(9493):1267- 1278.
    • (2005) Lancet , vol.366 , Issue.9493 , pp. 1267-1278
    • Baigent, C.1    Keech, A.2    Kearney, P.M.3
  • 12
    • 79959332777 scopus 로고    scopus 로고
    • Efficacy of statins in familial hypercholesterolaemia: A long term cohort study
    • Versmissen J, Oosterveer DM, Yazdanpanah M, et al. Efficacy of statins in familial hypercholesterolaemia: a long term cohort study. BMJ. 2008;337:a2423.
    • (2008) BMJ , vol.337
    • Versmissen, J.1    Oosterveer, D.M.2    Yazdanpanah, M.3
  • 13
    • 81355160365 scopus 로고    scopus 로고
    • Reduction in mortality in subjects with homozygous familial hypercholesterolemia associated with advances in lipid-lowering therapy
    • Raal FJ, Pilcher GJ, Panz VR, et al. Reduction in mortality in subjects with homozygous familial hypercholesterolemia associated with advances in lipid-lowering therapy. Circulation. 2011;124(20):2202-2207.
    • (2011) Circulation. , vol.124 , Issue.20 , pp. 2202-2207
    • Raal, F.J.1    Pilcher, G.J.2    Panz, V.R.3
  • 14
    • 79959728614 scopus 로고    scopus 로고
    • Effect of lipid-lowering treatment on natural history of heterozygous familial hypercholesterolemia in past three decades
    • Elis A, Zhou R, Stein EA. Effect of lipid-lowering treatment on natural history of heterozygous familial hypercholesterolemia in past three decades. Am J Cardiol. 2011;108(2):223-226.
    • (2011) Am J Cardiol. , vol.108 , Issue.2 , pp. 223-226
    • Elis, A.1    Zhou, R.2    Stein, E.A.3
  • 15
    • 77952525229 scopus 로고    scopus 로고
    • Colesevelam added to combination therapy with a statin and ezetimibe in patients with familial hypercholesterolemia: A 12-week, multicenter, randomized, double-blind, controlled trial
    • Huijgen R, Abbink EJ, Bruckert E, et al. Colesevelam added to combination therapy with a statin and ezetimibe in patients with familial hypercholesterolemia: a 12-week, multicenter, randomized, double-blind, controlled trial. Clin Ther. 2010;32(4):615-625.
    • (2010) Clin Ther. , vol.32 , Issue.4 , pp. 615-625
    • Huijgen, R.1    Abbink, E.J.2    Bruckert, E.3
  • 16
    • 78649390237 scopus 로고    scopus 로고
    • Lipoprotein apheresis
    • Thompson GR. Lipoprotein apheresis. Curr Opin Lipidol. 2010;21(6):487-491.
    • (2010) Curr Opin Lipidol. , vol.21 , Issue.6 , pp. 487-491
    • Thompson, G.R.1
  • 17
    • 74749107585 scopus 로고    scopus 로고
    • Efficacy criteria and cholesterol targets for LDL apheresis
    • Thompson GR, Barbir M, Davies D, et al. Efficacy criteria and cholesterol targets for LDL apheresis. Atherosclerosis. 2010;208(2):317-321.
    • (2010) Atherosclerosis , vol.208 , Issue.2 , pp. 317-321
    • Thompson, G.R.1    Barbir, M.2    Davies, D.3
  • 18
    • 84870468359 scopus 로고    scopus 로고
    • American Society For Apheresis. Available from: Accessed April 24, 2012
    • American Society For Apheresis. Therapeutic apheresis: a guide to billing and securing appropriate reimbursement, 2006. Available from: http://www.apheresis.org/~ASSETS/DOCUMENT/PDF/Committee/Therapeutic%20Apheresis- A%20Guide%20to%20Billing%20and%20Securing%20Appropriate%20Reimbursement%20(2006) .pdf. Accessed April 24, 2012.
    • (2006) Therapeutic Apheresis: A Guide to Billing and Securing Appropriate Reimbursement
  • 19
    • 79955000769 scopus 로고    scopus 로고
    • Liver transplantation as a treatment option for three siblings with homozygous familial hypercholesterolemia
    • Kucukkartallar T, Yankol Y, Kanmaz T, Topaloglu S, Acarli K, Kalayoglu M. Liver transplantation as a treatment option for three siblings with homozygous familial hypercholesterolemia. Pediatr Transplant. 2011;15(3):281-284.
    • (2011) Pediatr Transplant. , vol.15 , Issue.3 , pp. 281-284
    • Kucukkartallar, T.1    Yankol, Y.2    Kanmaz, T.3    Topaloglu, S.4    Acarli, K.5    Kalayoglu, M.6
  • 20
    • 79952516166 scopus 로고    scopus 로고
    • Drugs in development for management of lipoprotein disorders
    • Brown WV, Bays H, Davidson M, Goldberg A. Drugs in development for management of lipoprotein disorders. J Clin Lipidol. 2011;5(2):66-75.
    • (2011) J Clin Lipidol. , vol.5 , Issue.2 , pp. 66-75
    • Brown, W.V.1    Bays, H.2    Davidson, M.3    Goldberg, A.4
  • 21
    • 67649834056 scopus 로고    scopus 로고
    • A proprotein convertase subtilisin/kexin type 9 neutralizing antibody reduces serum cholesterol in mice and nonhuman primates
    • Chan JC, Piper DE, Cao Q, et al. A proprotein convertase subtilisin/kexin type 9 neutralizing antibody reduces serum cholesterol in mice and nonhuman primates. Proc Natl Acad Sci U S A. 2009;106(24): 9820-9825.
    • (2009) Proc Natl Acad Sci U S A , vol.106 , Issue.24 , pp. 9820-9825
    • Chan, J.C.1    Piper, D.E.2    Cao, Q.3
  • 22
    • 50149101511 scopus 로고    scopus 로고
    • Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates
    • Frank-Kamenetsky M, Grefhorst A, Anderson NN, et al. Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates. Proc Natl Acad Sci U S A. 2008;105(33):11915-11920.
    • (2008) Proc Natl Acad Sci U S A. , vol.105 , Issue.33 , pp. 11915-11920
    • Frank-Kamenetsky, M.1    Grefhorst, A.2    Anderson, N.N.3
  • 24
    • 79952049508 scopus 로고    scopus 로고
    • Effects of currently prescribed LDL-C-lowering drugs on PCSK9 and implications for the next generation of LDL-C-lowering agents
    • Konrad RJ, Troutt JS, Cao G. Effects of currently prescribed LDL-C-lowering drugs on PCSK9 and implications for the next generation of LDL-C-lowering agents. Lipids Health Dis. 2011;10:38.
    • (2011) Lipids Health Dis. , vol.10 , pp. 38
    • Konrad, R.J.1    Troutt, J.S.2    Cao, G.3
  • 25
    • 77949349469 scopus 로고    scopus 로고
    • Use of the thyroid hormone analogue eprotirome in statin-treated dyslipidemia
    • Ladenson PW, Kristensen JD, Ridgway EC, et al. Use of the thyroid hormone analogue eprotirome in statin-treated dyslipidemia. N Engl J Med. 2010;362(10):906-916.
    • (2010) N Engl J Med. , vol.362 , Issue.10 , pp. 906-916
    • Ladenson, P.W.1    Kristensen, J.D.2    Ridgway, E.C.3
  • 26
    • 42249115317 scopus 로고    scopus 로고
    • HDL metabolism and CETP inhibition
    • DOI 10.1097/CRD.0b013e31816a3b60, PII 0004541520080500000006
    • Barkowski RS, Frishman WH. HDL metabolism and CETP inhibition. Cardiol Rev. 2008;16(3):154-162. (Pubitemid 351549677)
    • (2008) Cardiology in Review , vol.16 , Issue.3 , pp. 154-162
    • Barkowski, R.S.1    Frishman, W.H.2
  • 27
    • 84856523563 scopus 로고    scopus 로고
    • Emerging therapies for dyslipidemia: Known knowns and known unknowns of MTP inhibitors
    • Raval SK, Raval PS, Jain MR. Emerging therapies for dyslipidemia: known knowns and known unknowns of MTP inhibitors. Recent Pat Endocr Metab Immune Drug Discov. 2012;6(1):24-29.
    • (2012) Recent Pat Endocr Metab Immune Drug Discov , vol.6 , Issue.1 , pp. 24-29
    • Raval, S.K.1    Raval, P.S.2    Jain, M.R.3
  • 28
    • 79959694696 scopus 로고    scopus 로고
    • A meta-analysis of low-density lipoprotein cholesterol, non-high-density lipoprotein cholesterol, and apolipoprotein B as markers of cardiovascular risk
    • Sniderman AD, Williams K, Contois JH, et al. A meta-analysis of low-density lipoprotein cholesterol, non-high-density lipoprotein cholesterol, and apolipoprotein B as markers of cardiovascular risk. Circ Cardiovasc Qual Outcomes. 2011;4(3):337-345.
    • (2011) Circ Cardiovasc Qual Outcomes , vol.4 , Issue.3 , pp. 337-345
    • Sniderman, A.D.1    Williams, K.2    Contois, J.H.3
  • 29
    • 77949512140 scopus 로고    scopus 로고
    • RNA targeting therapeutics: Molecular mechanisms of antisense oligonucleotides as a therapeutic platform
    • Bennett CF, Swayze EE. RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol. 2010;50:259-293.
    • (2010) Annu Rev Pharmacol Toxicol. , vol.50 , pp. 259-293
    • Bennett, C.F.1    Swayze, E.E.2
  • 30
    • 33750207033 scopus 로고    scopus 로고
    • Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B
    • DOI 10.1161/CIRCULATIONAHA.105.606442, PII 0000301720061017000012
    • Kastelein JJ, Wedel MK, Baker BF, et al. Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B. Circulation. 2006;114(16):1729-1735. (Pubitemid 44607121)
    • (2006) Circulation , vol.114 , Issue.16 , pp. 1729-1735
    • Kastelein, J.J.P.1    Wedel, M.K.2    Baker, B.F.3    Su, J.4    Bradley, J.D.5    Yu, R.Z.6    Chuang, E.7    Graham, M.J.8    Crooke, R.M.9
  • 31
    • 57449103966 scopus 로고    scopus 로고
    • Lack of pharmacokinetic interaction of mipomersen sodium (ISIS 301012), a 2′-O-methoxyethyl modified antisense oligonucleotide targeting apolipoprotein B100 messenger RNA, with simvastatin and ezetimibe
    • Yu RZ, Geary RS, Flaim JD, et al. Lack of pharmacokinetic interaction of mipomersen sodium (ISIS 301012), a 2′-O-methoxyethyl modified antisense oligonucleotide targeting apolipoprotein B100 messenger RNA, with simvastatin and ezetimibe. Clin Pharmacokinet. 2009;48(1):39-50.
    • (2009) Clin Pharmacokinet. , vol.48 , Issue.1 , pp. 39-50
    • Yu, R.Z.1    Geary, R.S.2    Flaim, J.D.3
  • 32
    • 77951069859 scopus 로고    scopus 로고
    • Effect of mipomersen, an apolipoprotein B synthesis inhibitor, on low-density lipoprotein cholesterol in patients with familial hypercholesterolemia
    • Akdim F, Visser ME, Tribble DL, et al. Effect of mipomersen, an apolipoprotein B synthesis inhibitor, on low-density lipoprotein cholesterol in patients with familial hypercholesterolemia. Am J Cardiol. 2010;105(10):1413- 1419.
    • (2010) Am J Cardiol. , vol.105 , Issue.10 , pp. 1413-1419
    • Akdim, F.1    Visser, M.E.2    Tribble, D.L.3
  • 33
    • 77950332314 scopus 로고    scopus 로고
    • Efficacy and safety of mipomersen, an antisense inhibitor of apolipoprotein B, in hypercholesterolemic subjects receiving stable statin therapy
    • Akdim F, Stroes ES, Sijbrands EJ, et al. Efficacy and safety of mipomersen, an antisense inhibitor of apolipoprotein B, in hypercholesterolemic subjects receiving stable statin therapy. J Am Coll Cardiol. 2010;55(15):1611-1618.
    • (2010) J Am Coll Cardiol , vol.55 , Issue.15 , pp. 1611-1618
    • Akdim, F.1    Stroes, E.S.2    Sijbrands, E.J.3
  • 34
    • 79959661095 scopus 로고    scopus 로고
    • Efficacy of apolipoprotein B synthesis inhibition in subjects with mild-to-moderate hyperlipidaemia
    • Akdim F, Tribble DL, Flaim JD, et al. Efficacy of apolipoprotein B synthesis inhibition in subjects with mild-to-moderate hyperlipidaemia. Eur Heart J. 2011;32(21):2650-2659.
    • (2011) Eur Heart J. , vol.32 , Issue.21 , pp. 2650-2659
    • Akdim, F.1    Tribble, D.L.2    Flaim, J.D.3
  • 35
    • 82755163631 scopus 로고    scopus 로고
    • Safety and efficacy of mipomersen administered as add-on therapy in patients with hypercholesterolemia and high cardiovascular risk
    • Abstract
    • Cromwell WC, Thomas GS, Boltje I, Chin W, Davidson M. Safety and efficacy of mipomersen administered as add-on therapy in patients with hypercholesterolemia and high cardiovascular risk. J Am Coll Cardiol. 2011:Abstract.
    • (2011) J Am Coll Cardiol
    • Cromwell, W.C.1    Thomas, G.S.2    Boltje, I.3    Chin, W.4    Davidson, M.5
  • 36
    • 77949485460 scopus 로고    scopus 로고
    • Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: A randomised, double-blind, placebo-controlled trial
    • Raal FJ, Santos RD, Blom DJ, et al. Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Lancet. 2010;375(9719):998-1006.
    • (2010) Lancet. , vol.375 , Issue.9719 , pp. 998-1006
    • Raal, F.J.1    Santos, R.D.2    Blom, D.J.3
  • 37
    • 82755198552 scopus 로고    scopus 로고
    • A randomized, double-blind, placebo-controlled study to assess efficacy and safety of mipomersen as add-on-therapy in herterozygous familial hyoercholesterolemia patients wirh coronary artery disease
    • Abstract
    • Stein E, Dufour R, Gagne C, et al. A randomized, double-blind, placebo-controlled study to assess efficacy and safety of mipomersen as add-on-therapy in herterozygous familial hyoercholesterolemia patients wirh coronary artery disease. Eur Heart J. 2010;31 Suppl 1:Abstract.
    • (2010) Eur Heart J , vol.31 , Issue.SUPPL. 1
    • Stein, E.1    Dufour, R.2    Gagne, C.3
  • 38
    • 82755190754 scopus 로고    scopus 로고
    • Apolipoprotein B-synthesis inhibition by mipomersen reduces LDL-C when added to maximally tolerated lipid-lowering medication in patients with severe heterozygous hypercholesterolemia
    • Abstract
    • Tardif JC, Ceska R, Burgess LJ, et al. Apolipoprotein B-synthesis inhibition by mipomersen reduces LDL-C when added to maximally tolerated lipid-lowering medication in patients with severe heterozygous hypercholesterolemia. J Am Coll Cardiol. 2011:Abstract.
    • (2011) J Am Coll Cardiol
    • Tardif, J.C.1    Ceska, R.2    Burgess, L.J.3
  • 39
    • 84860615159 scopus 로고    scopus 로고
    • Mipomersen, an apolipoprotein B synthesis inhibitor, lowers low-density lipoprotein cholesterol in high-risk statin-intolerant patients: A randomized, double-blind, placebo-controlled trial
    • April 16, Epub ahead of print
    • Visser ME, Wagener G, Baker BF, et al. Mipomersen, an apolipoprotein B synthesis inhibitor, lowers low-density lipoprotein cholesterol in high-risk statin-intolerant patients: a randomized, double-blind, placebo-controlled trial. Eur Heart J. April 16, 2012. [Epub ahead of print.]
    • (2012) Eur Heart J
    • Visser, M.E.1    Wagener, G.2    Baker, B.F.3
  • 40
    • 77951072532 scopus 로고    scopus 로고
    • Effect of apolipoprotein-B synthesis inhibition on liver triglyceride content in patients with familial hypercholesterolemia
    • Visser ME, Akdim F, Tribble DL, et al. Effect of apolipoprotein-B synthesis inhibition on liver triglyceride content in patients with familial hypercholesterolemia. J Lipid Res. 2010;51(5):1057-1062.
    • (2010) J Lipid Res. , vol.51 , Issue.5 , pp. 1057-1062
    • Visser, M.E.1    Akdim, F.2    Tribble, D.L.3
  • 41
    • 82755198550 scopus 로고    scopus 로고
    • 760 mipomersen and apoB synthesis inhibitor evaluation of potential to reduce necessity for lipid-apheresis in patients with heterozygous FH and CAD
    • Parhofer KG, Vogt A. 760 mipomersen and apoB synthesis inhibitor evaluation of potential to reduce necessity for lipid-apheresis in patients with heterozygous FH and CAD. Atherosclerosis. 2011;12(1):159.
    • (2011) Atherosclerosis. , vol.12 , Issue.1 , pp. 159
    • Parhofer, K.G.1    Vogt, A.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.