Long-term mipomersen treatment is associated with a reduction in cardiovascular events in patients with familial hypercholesterolemia

Journal of Clinical Lipidology

Volumn 10, Issue 4, 2016, Pages 1011-1021

  Duell, P Barton ^a   Santos, Raul D ^b   Kirwan, Bridget Anne ^c,e   Witztum, Joseph L ^e   Tsimikas, Sotirios ^f,g   Kastelein, John J P ^d  

^a OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^b UNIVERSITY OF SÃO PAULO   (Brazil)

^c SOCAR RESEARCH SA   (Switzerland)

^d LONDON SCHOOL OF HYGIENE AND TROPICAL MEDICINE   (United Kingdom)

^e UNIVERSITY OF CALIFORNIA   (United States)

^f ISIS PHARMACEUTICALS   (United States)

^g UNIVERSITY OF AMSTERDAM   (Netherlands)

Author keywords

Antisense; Familial hypercholesterolemia; LDL Cholesterol; Major adverse cardiovascular events; Mipomersen

Indexed keywords

LIPOPROTEIN A; LOW DENSITY LIPOPROTEIN; MIPOMERSEN; LIPID; OLIGONUCLEOTIDE;

ADULT; ARTICLE; BRAIN ISCHEMIA; FAMILIAL HYPERCHOLESTEROLEMIA; FEMALE; HEART DEATH; HEART DISEASE; HEART INFARCTION; HEART MUSCLE REVASCULARIZATION; HUMAN; LONG TERM CARE; MALE; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); UNSTABLE ANGINA PECTORIS; BLOOD; CARDIOVASCULAR DISEASES; COMPLICATION; CONTROLLED STUDY; HYPERLIPOPROTEINEMIA TYPE II; MIDDLE AGED; RANDOMIZED CONTROLLED TRIAL; TIME FACTOR;

ADULT; CARDIOVASCULAR DISEASES; FEMALE; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; LIPIDS; MALE; MIDDLE AGED; OLIGONUCLEOTIDES; TIME FACTORS;

EID: 84978529098     PISSN: 19332874     EISSN: 18764789     Source Type: Journal    
DOI: 10.1016/j.jacl.2016.04.013     Document Type: Article

References (29)

1. 1 Hopkins, P.N., Toth, P.P., Ballantyne, C.M., Rader, D.J., Familial hypercholesterolemias: prevalence, genetics, diagnosis and screening recommendations from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Clin Lipidol 5:3 Suppl (2011), S9–S17.
2. 2 Alonso, R., Andres, E., Mata, N., et al. Lipoprotein(a) levels in familial hypercholesterolemia: an important predictor of cardiovascular disease independent of the type of LDL receptor mutation. J Am Coll Cardiol 63:19 (2014), 1982–1989.
3. 3 Raal, F.J., Pilcher, G.J., Panz, V.R., et al. Reduction in mortality in subjects with homozygous familial hypercholesterolemia associated with advances in lipid-lowering therapy. Circulation 124:20 (2011), 2202–2207.
4. 4 Tietge, U.J., Kozarsky, K.F., Donahee, M.H., Rader, D.J., A tetracycline-regulated adenoviral expression system for in vivo delivery of transgenes to lung and liver. J Gene Med 5:7 (2003), 567–575.
5. 5 Raal, F.J., Santos, R.D., Blom, D.J., 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 375:9719 (2010), 998–1006.
6. 6 McGowan, M.P., Tardif, J.C., Ceska, R., et al. Randomized, placebo-controlled trial of mipomersen in patients with severe hypercholesterolemia receiving maximally tolerated lipid-lowering therapy. PLoS One, 7(11), 2012, e49006.
7. 7 Stein, E.A., Dufour, R., Gagne, C., et al. Apolipoprotein B synthesis inhibition with mipomersen in heterozygous familial hypercholesterolemia: results of a randomized, double-blind, placebo-controlled trial to assess efficacy and safety as add-on therapy in patients with coronary artery disease. Circulation 126:19 (2012), 2283–2292.
8. 8 Thomas, G.S., Cromwell, W.C., Ali, S., Chin, W., Flaim, J.D., Davidson, M., Mipomersen, an apolipoprotein B synthesis inhibitor, reduces atherogenic lipoproteins in patients with severe hypercholesterolemia at high cardiovascular risk: a randomized, double-blind, placebo-controlled trial. J Am Coll Cardiol 62:23 (2013), 2178–2184.
9. 9 Santos, R.D., Duell, P.B., East, C., et al. Long-term efficacy and safety of mipomersen in patients with familial hypercholesterolaemia: 2-year interim results of an open-label extension. Eur Heart J 36:9 (2015), 566–575.
10. 10 Santos, R.D., Raal, F.J., Catapano, A.L., Witztum, J.L., Steinhagen-Thiessen, E., Tsimikas, S., Mipomersen, an antisense oligonucleotide to apolipoprotein B-100, reduces lipoprotein(a) in various populations with hypercholesterolemia: results of 4 phase III trials. Arterioscler Thromb Vasc Biol 35:3 (2015), 689–699.
11. 11 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 376:9753 (2010), 1670–1681.
12. 12 Thygesen, K., Alpert, J.S., Jaffe, A.S., et al. Third universal definition of myocardial infarction. J Am Coll Cardiol 60:16 (2012), 1581–1598.
13. 13 Hobbs, H.H., Brown, M.S., Russell, D.W., Davignon, J., Goldstein, J.L., Deletion in the gene for the low-density-lipoprotein receptor in a majority of French Canadians with familial hypercholesterolemia. N Engl J Med 317:12 (1987), 734–737.
14. 14 Steinberg, D., Glass, C.K., Witztum, J.L., Evidence mandating earlier and more aggressive treatment of hypercholesterolemia. Circulation 118:6 (2008), 672–677.
15. 15 Robinson, J.G., Kastelein, J.J., PCSK9 Inhibitors and Cardiovascular Events. N Engl J Med, 373(8), 2015, 774.
16. 16 Sabatine, M.S., Giugliano, R.P., Wiviott, S.D., et al. Efficacy and safety of evolocumab in reducing lipids and cardiovascular events. N Engl J Med 372:16 (2015), 1500–1509.
17. 17 Albers, J.J., Slee, A., O'Brien, K.D., et al. Relationship of apolipoproteins A-1 and B, and lipoprotein(a) to cardiovascular outcomes: the AIM-HIGH trial (Atherothrombosis Intervention in Metabolic Syndrome with Low HDL/High Triglyceride and Impact on Global Health Outcomes). J Am Coll Cardiol 62:17 (2013), 1575–1579.
18. 18 Khera, A.V., Everett, B.M., Caulfield, M.P., et al. Lipoprotein(a) concentrations, rosuvastatin therapy, and residual vascular risk: an analysis from the JUPITER Trial (Justification for the Use of Statins in Prevention: an Intervention Trial Evaluating Rosuvastatin). Circulation 129:6 (2014), 635–642.
19. 19 The Lipid Research Clinics Coronary Primary Prevention Trial results. I. Reduction in incidence of coronary heart disease. JAMA 251:3 (1984), 351–364.
20. 20 Buchwald, H., Varco, R.L., Matts, J.P., et al. Effect of partial ileal bypass surgery on mortality and morbidity from coronary heart disease in patients with hypercholesterolemia. Report of the Program on the Surgical Control of the Hyperlipidemias (POSCH). N Engl J Med 323:14 (1990), 946–955.
21. 21 Cannon, C.P., Blazing, M.A., Giugliano, R.P., et al. Ezetimibe Added to Statin Therapy after Acute Coronary Syndromes. N Engl J Med 372:25 (2015), 2387–2397.
22. 22 Canner, P.L., Berge, K.G., Wenger, N.K., et al. Fifteen year mortality in Coronary Drug Project patients: long-term benefit with niacin. J Am Coll Cardiol 8:6 (1986), 1245–1255.
23. 23 Ridker, P.M., Danielson, E., Fonseca, F.A., et al. Rosuvastatin to prevent vascular events in men and women with elevated C-reactive protein. N Engl J Med 359:21 (2008), 2195–2207.
24. 24 Jarcho, J.A., Keaney, J.F. Jr., Proof that lower is better–LDL cholesterol and IMPROVE-IT. N Engl J Med 372:25 (2015), 2448–2450.
25. 25 Versmissen, J., Oosterveer, D.M., Yazdanpanah, M., et al. Efficacy of statins in familial hypercholesterolaemia: a long term cohort study. BMJ, 337, 2008, a2423.
26. 26 Mabuchi, H., Koizumi, J., Shimizu, M., et al. Long-term efficacy of low-density lipoprotein apheresis on coronary heart disease in familial hypercholesterolemia. Hokuriku-FH-LDL-Apheresis Study Group. Am J Cardiol 82:12 (1998), 1489–1495.
27. 27 Leebmann, J., Roeseler, E., Julius, U., et al. Lipoprotein apheresis in patients with maximally tolerated lipid-lowering therapy, lipoprotein(a)-hyperlipoproteinemia, and progressive cardiovascular disease: prospective observational multicenter study. Circulation 128:24 (2013), 2567–2576.
28. 28 Rosada, A., Kassner, U., Vogt, A., Willhauck, M., Parhofer, K., Steinhagen-Thiessen, E., Does regular lipid apheresis in patients with isolated elevated lipoprotein(a) levels reduce the incidence of cardiovascular events?. Artif Organs 38:2 (2014), 135–141.
29. 29 Arai, K., Orsoni, A., Mallat, Z., et al. Acute impact of apheresis on oxidized phospholipids in patients with familial hypercholesterolemia. J Lipid Res 53:8 (2012), 1670–1678.