Pharmacokinetic and pharmacodynamic assessment of alirocumab in patients with familial hypercholesterolemia associated with proprotein convertase subtilisin/kexin type 9 gain-of-function or apolipoprotein B loss-of-function mutations

Journal of Clinical Lipidology - Tập 13 - Trang 970-978 - 2019
Paul N. Hopkins1, Michel Krempf2, Eric Bruckert3, Stephen Donahue4, Feng Yang4, Yi Zhang4, A. Thomas DiCioccio4
1School of Medicine, University of Utah, Salt Lake City, UT, USA
2CHU de Nantes – Hôpital Nord Laennec, Saint-Herblain, France
3Hôpital de La Pitié-Salpêtrière, Paris, France
4Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA

Tài liệu tham khảo

Santos, 2016, Defining severe familial hypercholesterolaemia and the implications for clinical management: a consensus statement from the International Atherosclerosis Society Severe Familial Hypercholesterolemia Panel, Lancet Diabetes Endocrinol, 4, 850, 10.1016/S2213-8587(16)30041-9 Nordestgaard, 2013, Familial hypercholesterolaemia is underdiagnosed and undertreated in the general population: guidance for clinicians to prevent coronary heart disease: consensus statement of the European Atherosclerosis Society, Eur Heart J, 34, 3478, 10.1093/eurheartj/eht273 Sjouke, 2015, Homozygous autosomal dominant hypercholesterolaemia in the Netherlands: prevalence, genotype-phenotype relationship, and clinical outcome, Eur Heart J, 36, 560, 10.1093/eurheartj/ehu058 Hopkins, 2015, Characterization of autosomal dominant hypercholesterolemia caused by PCSK9 gain of function mutations and its specific treatment with alirocumab, a PCSK9 monoclonal antibody, Circ Cardiovasc Genet, 8, 823, 10.1161/CIRCGENETICS.115.001129 Defesche, 2017, Familial hypercholesterolaemia, Nat Rev Dis Primers, 3, 17093, 10.1038/nrdp.2017.93 Andersen, 2016, Familial defective apolipoprotein B-100: a review, J Clin Lipidol, 10, 1297, 10.1016/j.jacl.2016.09.009 Sjouke, 2015, Homozygous autosomal dominant hypercholesterolaemia: prevalence, diagnosis, and current and future treatment perspectives, Curr Opin Lipidol, 26, 200, 10.1097/MOL.0000000000000179 Borén, 2001, The molecular mechanism for the genetic disorder familial defective apolipoprotein B100, J Biol Chem, 276, 9214, 10.1074/jbc.M008890200 Shapiro, 2018, PCSK9: from basic science discoveries to clinical trials, Circ Res, 122, 1420, 10.1161/CIRCRESAHA.118.311227 Seidah, 2014, PCSK9: a key modulator of cardiovascular health, Circ Res, 114, 1022, 10.1161/CIRCRESAHA.114.301621 Horton, 2009, PCSK9: a convertase that coordinates LDL catabolism, J Lipid Res, 50, S172, 10.1194/jlr.R800091-JLR200 Stein, 2012, Effect of a monoclonal antibody to PCSK9 on LDL cholesterol, N Engl J Med, 366, 1108, 10.1056/NEJMoa1105803 Seidah, 2017, The PCSK9 revolution and the potential of PCSK9-based therapies to reduce LDL-cholesterol, Glob Cardiol Sci Pract, 2017, e201702 Roth, 2014, Alirocumab for hyperlipidemia: physiology of PCSK9 inhibition, pharmacodynamics and Phase I and II clinical trial results of a PCSK9 monoclonal antibody, Future Cardiol, 10, 183, 10.2217/fca.13.107 Rey, 2016, Relationship between low-density lipoprotein cholesterol, free proprotein convertase subtilisin/kexin type 9, and alirocumab levels after different lipid-lowering strategies, J Am Heart Assoc, 5, e003323, 10.1161/JAHA.116.003323 Reyes-Soffer, 2017, Effects of PCSK9 inhibition with alirocumab on lipoprotein metabolism in healthy humans, Circulation, 135, 352, 10.1161/CIRCULATIONAHA.116.025253 Giunzioni, 2015, New developments in atherosclerosis: clinical potential of PCSK9 inhibition, Vasc Health Risk Manag, 11, 493 Glerup, 2017, Physiological and therapeutic regulation of PCSK9 activity in cardiovascular disease, Basic Res Cardiol, 112, 32, 10.1007/s00395-017-0619-0 Abifadel, 2014, Living the PCSK9 adventure: from the identification of a new gene in familial hypercholesterolemia towards a potential new class of anticholesterol drugs, Curr Atheroscler Rep, 16, 439, 10.1007/s11883-014-0439-8 Friedewald, 1972, Estimation of the concentration of low-density lipoprotein cholesterol in plasma, without use of the preparative ultracentrifuge, Clin Chem, 18, 499, 10.1093/clinchem/18.6.499 Defesche, 2017, Efficacy of alirocumab in 1191 patients with a wide spectrum of mutations in genes causative for familial hypercholesterolemia, J Clin Lipidol, 11, 1338, 10.1016/j.jacl.2017.08.016 Raal, 2015, Inhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA Part B): a randomised, double-blind, placebo-controlled trial, Lancet, 385, 341, 10.1016/S0140-6736(14)61374-X Hartgers, 2018, Alirocumab efficacy in patients with double heterozygous, compound heterozygous, or homozygous familial hypercholesterolemia, J Clin Lipidol, 12, 390, 10.1016/j.jacl.2017.12.008 Kastelein, 2015, ODYSSEY FH I and FH II: 78 week results with alirocumab treatment in 735 patients with heterozygous familial hypercholesterolaemia, Eur Heart J, 36, 2996 McKenney, 2012, Safety and efficacy of a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 serine protease, SAR236553/REGN727, in patients with primary hypercholesterolemia receiving ongoing stable atorvastatin therapy, J Am Coll Cardiol, 59, 2344, 10.1016/j.jacc.2012.03.007 Brown, 2018, Retrospective on cholesterol homeostasis: the central role of Scap, Annu Rev Biochem, 87, 783, 10.1146/annurev-biochem-062917-011852 Tavori, 2015, On the function and homeostasis of PCSK9: reciprocal interaction with LDLR and additional lipid effects, Atherosclerosis, 238, 264, 10.1016/j.atherosclerosis.2014.12.017 Soria, 1989, Association between a specific apolipoprotein B mutation and familial defective apolipoprotein B-100, Proc Natl Acad Sci U S A, 86, 587, 10.1073/pnas.86.2.587 Guo, 2014, PCSK9 and lipid lowering drugs, Clin Chim Acta, 437, 66, 10.1016/j.cca.2014.07.008 Mayne, 2008, Plasma PCSK9 levels are significantly modified by statins and fibrates in humans, Lipids Health Dis, 7, 22, 10.1186/1476-511X-7-22 Cunningham, 2007, Structural and biophysical studies of PCSK9 and its mutants linked to familial hypercholesterolemia, Nat Struct Mol Biol, 14, 413, 10.1038/nsmb1235 Roth, 2012, Atorvastatin with or without an antibody to PCSK9 in primary hypercholesterolemia, N Engl J Med, 367, 1891, 10.1056/NEJMoa1201832 Gaudet, 2017, Effect of alirocumab on lipoprotein(a) over >/=1.5 years (from the Phase 3 ODYSSEY program), Am J Cardiol, 119, 40, 10.1016/j.amjcard.2016.09.010