Removal of plasma mature and furin cleaved proprotein convertase subtilisin/kexin 9 by low density lipoprotein apheresis in familial hypercholesterolemia: Development and application of a new assay for PCSK9

Journal of Clinical Endocrinology and Metabolism

Volumn 100, Issue 1, 2015, Pages E41-E49

  Hori, Mika ^a   Ishihara, Mitsuaki ^b   Yuasa, Yumiko ^a   Makino, Hisashi ^a   Yanagi, Koji ^c   Tamanaha, Tamiko ^a   Kishimoto, Ichiro ^a   Kujiraoka, Takeshi ^b   Hattori, Hiroaki ^b   Harada Shiba, Mariko ^a  

^a NATIONAL CEREBRAL AND CARDIOVASCULAR CENTER   (Japan)

^b BML   (Japan)

^c Osaka Chuo Byoin   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

APOLIPOPROTEIN B; CELLULOSE; DEXTRAN SULFATE; FURIN; LOW DENSITY LIPOPROTEIN CHOLESTEROL; MONOCLONAL ANTIBODY; PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9; SERINE PROTEINASE; UNCLASSIFIED DRUG; PCSK9 PROTEIN, HUMAN;

ADSORPTION KINETICS; APHERESIS; ARTICLE; CLINICAL ARTICLE; CONTROLLED STUDY; ENZYME ASSAY; ENZYME LINKED IMMUNOSORBENT ASSAY; FAMILIAL HYPERCHOLESTEROLEMIA; GEL FILTRATION CHROMATOGRAPHY; HUMAN; IMMUNOPRECIPITATION; LOW DENSITY LIPOPROTEIN APHERESIS; PROCESS DEVELOPMENT; PROTEIN BLOOD LEVEL; PROTEIN CLEAVAGE; PROTEIN TARGETING; SANDWICH ENZYME LINKED IMMUNOSORBENT ASSAY; STANDARDIZATION; ADULT; AGED; BLOOD; CROSSOVER PROCEDURE; FEMALE; HYPERLIPOPROTEINEMIA TYPE II; MALE; MIDDLE AGED; PROCEDURES; VERY ELDERLY; YOUNG ADULT;

ADULT; AGED; AGED, 80 AND OVER; APOLIPOPROTEINS B; BLOOD COMPONENT REMOVAL; CHOLESTEROL, LDL; CROSS-OVER STUDIES; ENZYME-LINKED IMMUNOSORBENT ASSAY; FEMALE; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; MALE; MIDDLE AGED; PROPROTEIN CONVERTASES; SERINE ENDOPEPTIDASES; YOUNG ADULT;

EID: 84920576491     PISSN: 0021972X     EISSN: 19457197     Source Type: Journal    
DOI: 10.1210/jc.2014-3066     Document Type: Article

References (35)

1. Goldstein JL, Hobbs HH, Brown MS. Familial hypercholesterolemia. In: Scriver CR BA, Sly WS, Valle D, eds. The Metabolic and Molecular Bases of Inherited Disease. 8th ed. New York: McGraw-Hill; 2001.
2. Abifadel M, Varret M, Rabés JP, et al. Mutations in PCSK9 cause autosomal dominant hypercholesterolemia. Nature Genetics. 2003; 34:154-156.
3. Benjannet S, Rhainds D, Essalmani R, et al. NARC-1/PCSK9 and its natural mutants: zymogen cleavage and effects on the low density lipoprotein (LDL) receptor andLDLcholesterol. J Biol Ch em. 2004; 279:48865-48875.
4. Maxwell KN,Breslow JL. Adenoviral-mediated expression of Pcsk9 in mice results in a low-density lipoprotein receptor knockout phenotype Proc Natl Acad Sci USA. 2004; 101 7100-7105.
5. Dubuc G, Tremblay M, ParéG, et al. A new method for measurement of total plasma PCSK9: clinical applications. J Lipid Res. 2010; 51:140-149.
6. Alborn WE, Cao G, Careskey HE, et al. Serum proprotein convertase subtilisin kexin type 9 is correlated directly with serum LDL cholesterol. Clin Chem. 2007;53:1814-1819.
7. Be njannet S, Rhainds D, Hamelin J, Nassoury N, Seidah NG. The proprotein convertase (PC) PCSK9 is inactivated by furin and/or PC5/6A: functional consequences of natural mutations and posttranslational modifications. J Biol Chem. 2006;281:30561-30572.
8. Essalmani R, Susan-Resiga D, Chamberland A, et al. In vivo evidence that furin from hepatocytes inactivates PCSK9. J Biol Chem. 2011;286:4257-4263.
9. Han B, Eacho PI, Knierman MD, et al. Isolation and characterization of the circulating truncated form of PCSK9. J Lipid Res. 2014; 55:1505-1514.
10. Lipari MT, Li W, Moran P , et al. Furin-cleaved proprotein convertase subtilisin/kexin type 9 (PCSK9) is active and modulates low density lipoprotein receptor and serum cholesterol levels. J Biol Chem. 2012;287:43482-43491.
11. Yokoyama S, Hayashi R, Satani M, Yamamoto A. Selective removal of low density lipoprotein by plasmapheresis in familial hypercholesterolemia. Arteriosclerosis. 1985;5:613-622.
12. Makino H, Harada-Shiba M. Long-term effect of low-density lipoprotein apheresis in patients with homozygous familial hypercholesterolemia. Ther Apher Dial. 2003;7:397-401.
13. Kobayashi J, Katsube S, Shimoda M , et al. Single LDL apheresis improves serum remnant-like particle-cholesterol, C-reactive protein, and malondialdehyde-modified-low-density lipoprotein concentrations in Japanese hypercholesterolemic subjects. Clin Chim Acta. 2002;321:107-112.
14. Kojima S, Harada-Shiba M, Toyota Y, et al. Changes in coagulation factors by passage through a dextran sulfate cellulose column during low-density lipoprotein apheresis. Int J Artif Organs. 1992 ;15:185-190.
15. Yuasa Y, Osaki T, Makino H, et al. Proteomic analysis of proteins eliminated by low-density lipoprotein apheresis. Ther Apher Dial. 2014;18:93-102.
16. Tavori H, Giunzioni I, Linton MF, Fazio S. Loss of plasma proprotein convertase subtilisin/kexin 9 (PCSK9) after lipoprotein apheresis. Circ Res. 2013;113:1290-1295.
17. Cameron J, Bogsrud MP, Tveten K, et al. Serum levels of proprotein convertase subtilisin/kexin type 9 in subjects with familial hypercholesterolemia indicate that proprotein convertase subtilisin/kexin type 9 is cleared from plasma by low-density lipop rotein receptorindependent pathways. Transl Res. 2012;160:125-130.
18. Mabuchi H, Miyamoto S, Ueda K, et al. Causes of death in patients with familial hypercholesterolemia. Atherosclerosis. 1986;61:1-6.
19. Harada-Shiba M, Arai H, Oikawa S, et al. Guidelines for the management of familial hypercholesterolemia. J Atheroscler Thromb. 2012;19:1043-1060.
20. Harada-ShibaM,Takagi A, Miyamoto Y, et al. Clinical features and genetic analysis of autosomal recessive hypercholesterolemia. J Clin Endocrinol Metab. 2003;88:2541-2547.
21. Ishihara M, Kujiraoka T, Iwasaki T, et al. A sandwich enzymelinked immunosorbent assay for human plasma apolipoprotein A-V concentration. J Lipid Res. 2005;46:2015-2022.
22. Oka T, Kujiraoka T, Ito M, et al. Distribution of ph ospholipid transfer protein in human plasma: presence of two forms of phospholipid transfer protein, one catalytically active and the other inactive. J Lipid Res. 2000;41:1651-1657.
23. Dubuc G, Chamberland A , Wassef H, et al. Statins upregulate PCSK9, the gene encoding the proprotein convertase neural apop-tosis-regulated convertase-1 implicated in familial hypercholesterolemia. Arterioscler Thromb Vasc Biol. 2004;24:1454-14 59.
24. Dong B, Wu M, Li H, et al. Strong induction of PCSK9 gene expression through HNF1alpha and SREBP2: mechanism for the resistance to LDL-cholesterol lowering effect of statins in dyslipidemic hamsters. J Li pid Res. 2010;51:1486-1495.
25. Frank-Kamenetsky M, Grefhorst A, Anderson NN, et al. Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman prim ates. Proc Natl Acad Sci USA. 2008;105:11915-11920.
26. Yamamoto T,Harada-Shiba M,Nakatani M,et al. Cholesterollowering action of BNA-based antisense oligonucleotides targeting PCSK9 in athero genic diet-induced hypercholesterolemic mice Mol Therapy Nucleic Acids. 2012 1 e22.
27. Chan JC, Piper DE, Cao Q, et al. A proprotein convertase subtilisin/kexin type 9 neutralizing antibody re duces serum cholesterol in mice and nonhuman primates. Proc Natl Acad SciUSA. 2009;106:9820-9825.
28. Stein EA, Gipe D, Bergeron J, et al. Effect of a monoclonal antibody to PCSK9, REGN727/SAR236 553, to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolaemia on stable statin dose with or without ezetimibe therapy: a phase 2 randomised controlled trial. Lancet. 2012;380:29-36.
29. Giugliano RP, Desai NR, Kohli P, et al. Efficacy, safety, and tolerability of a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 in combination with a statin in patients with hypercholesterolaem ia (LAPLACE-TIMI 57): a randomised, placebocontrolled, dose-ranging, phase 2 study. Lancet. 2012;380:2007-2017.
30. Nozue T, Hattori H, Ishihara M, et al. Comparison of effects of pitavastatin versus pravastatin on serum proprotein convertase subtilisin/kexin type 9 levels in statin-naïve patients with coronary artery disease. Am J Cardiol. 2013;111:1415-1419.
31. Kosenko T, Golder M, Leblond G, Weng W, Lagace TA. Low density lipoprotein binds to proprotein convertase subtilisin/kexin type-9 (PCSK9) in human plasma and inhibits PCSK9-mediated low density lipoprotein receptor degradation. J Biol Chem. 2013;288: 8279-8288.
32. Tavori H, Fan D, Blakemore JL, et al. Serum proprotein convertase subtilisin/kexin type 9 and cell surface low-density lipoprotein receptor: evidence for a reciprocal regulation. Circulation. 2013;127: 2403-2413.
33. Sun H, Samarghandi A, Zhang N, Yao Z, Xiong M, Teng BB. Proprotein c onvertase subtilisin/kexin type 9 interacts with apolipoprotein B and prevents its intracellular degradation, irrespective of the low-density lipoprotein receptor. Arterioscler Thromb Vasc Biol. 2012;32:1585-1595.
34. Fan D, Yancey PG, Qiu S, et al. Self-Association of human PCSK9 correlates with its LDLR-degrading activity. Biochemistry. 2008; 47:1631-1639.
35. Holzer M, Trieb M, Konya V, W adsack C, Heinemann A, Marsche G. Aging affects high-density lipoprotein composition and function. Biochim Biophys Acta. 2013;1831:1442-1448.