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References

  • 1
    Ahmed R, Pulendran B. Learning vaccinology from viral infections. J Exp Med 2011; 208: 23479.
  • 2
    Kasturi SP, Skountzou I, Albrecht RA, Koutsonanos D, Hua T, Nakaya HI, Ravindran R, Stewart S, Alam M, Kwissa M, Villinger F, Murthy N, Steel J, Jacob J, Hogan RJ, Garcia-Sastre A, Compans R, Pulendran B. Programming the magnitude and persistence of antibody responses with innate immunity. Nature 2011; 470: 5437.
  • 3
    Jahn EM, Schneider CK. How to systematically evaluate immunogenicity of therapeutic proteins – regulatory considerations. N Biotechnol 2009; 25: 2806.
  • 4
    Astermark J, Altisent C, Batorova A, Diniz MJ, Gringeri A, Holme PA, Karafoulidou A, Lopez-Fernandez MF, Reipert BM, Rocino A, Schiavoni M, von Depka M, Windyga J, Fijnvandraat K. Non-genetic risk factors and the development of inhibitors in haemophilia: a comprehensive review and consensus report. Haemophilia 2010; 16: 74766.
  • 5
    Pavlova A, Delev D, Lacroix-Desmazes S, Schwaab R, Mende M, Fimmers R, Astermark J, Oldenburg J. Impact of polymorphisms of the major histocompatibility complex class II, interleukin-10, tumor necrosis factor-alpha and cytotoxic T-lymphocyte antigen-4 genes on inhibitor development in severe hemophilia A. J Thromb Haemost 2009; 7: 200615.
  • 6
    Gouw SC, van den Berg HM. The multifactorial etiology of inhibitor development in hemophilia: genetics and environment. Semin Thromb Hemost 2009; 35: 72334.
  • 7
    Pratt KP. Inhibitory antibodies in hemophilia A. Curr Opin Hematol 2012; 19: 399405.
  • 8
    Kempton CL, White GC II. How we treat a hemophilia A patient with a factor VIII inhibitor. Blood 2009; 113: 1117.
  • 9
    Qian J, Collins M, Sharpe AH, Hoyer LW. Prevention and treatment of factor VIII inhibitors in murine hemophilia A. Blood 2000; 95: 13249.
  • 10
    Reipert BM, Sasgary M, Ahmad RU, Auer W, Turecek PL, Schwarz HP. Blockade of CD40/CD40 ligand interactions prevents induction of factor VIII inhibitors in hemophilic mice but does not induce lasting immune tolerance. Thromb Haemost 2001; 86: 134552.
  • 11
    Wu H, Reding M, Qian J, Okita DK, Parker E, Lollar P, Hoyer LW, Conti-Fine BM. Mechanism of the immune response to human factor VIII in murine hemophilia A. Thromb Haemost 2001; 85: 12533.
  • 12
    Ragni MV, Bontempo FA, Lewis JH. Disappearance of inhibitor to factor VIII in HIV-infected hemophiliacs with progression to AIDS or severe ARC. Transfusion 1989; 29: 4479.
  • 13
    Jones TD, Phillips WJ, Smith BJ, Bamford CA, Nayee PD, Baglin TP, Gaston JS, Baker MP. Identification and removal of a promiscuous CD4+ T cell epitope from the C1 domain of factor VIII. J Thromb Haemost 2005; 3: 9911000.
  • 14
    James EA, Kwok WW, Ettinger RA, Thompson AR, Pratt KP. T-cell responses over time in a mild hemophilia A inhibitor subject: epitope identification and transient immunogenicity of the corresponding self-peptide. J Thromb Haemost 2007; 5: 2399407.
  • 15
    James EA, van Haren SD, Ettinger RA, Fijnvandraat K, Liberman JA, Kwok WW, Voorberg J, Pratt KP. T-cell responses in two unrelated hemophilia A inhibitor subjects include an epitope at the factor VIII R593C missense site. J Thromb Haemost 2011; 9: 68999.
  • 16
    van den Brink EN, Bril WS, Turenhout EA, Zuurveld M, Bovenschen N, Peters M, Yee TT, Mertens K, Lewis DA, Ortel TL, Lollar P, Scandella D, Voorberg J. Two classes of germline genes both derived from the V(H)1 family direct the formation of human antibodies that recognize distinct antigenic sites in the C2 domain of factor VIII. Blood 2002; 99: 282834.
  • 17
    Jacquemin MG, Desqueper BG, Benhida A, Vander EL, Hoylaerts MF, Bakkus M, Thielemans K, Arnout J, Peerlinck K, Gilles JG, Vermylen J, Saint-Remy JM. Mechanism and kinetics of factor VIII inactivation: study with an IgG4 monoclonal antibody derived from a hemophilia A patient with inhibitor. Blood 1998; 92: 496506.
  • 18
    Dasgupta S, Navarrete AM, Bayry J, Delignat S, Wootla B, Andre S, Christophe O, Nascimbeni M, Jacquemin M, Martinez-Pomares L, Geijtenbeek TB, Moris A, Saint-Remy JM, Kazatchkine MD, Kaveri SV, Lacroix-Desmazes S. A role for exposed mannosylations in presentation of human therapeutic self-proteins to CD4+ T lymphocytes. Proc Natl Acad Sci USA 2007; 104: 896570.
  • 19
    Oldenburg J, Picard JK, Schwaab R, Brackmann HH, Tuddenham EG, Simpson E. HLA genotype of patients with severe haemophilia A due to intron 22 inversion with and without inhibitors of factor VIII. Thromb Haemost 1997; 77: 23842.
  • 20
    van Haren SD, Herczenik E, ten Brinke A, Mertens K, Voorberg J, Meijer AB. HLA-DR-presented peptide repertoires derived from human monocyte-derived dendritic cells pulsed with blood coagulation factor VIII. Mol Cell Proteomics 2011; 10: M110.002246.
  • 21
    Lollar P. Pathogenic antibodies to coagulation factors. Part one: factor VIII and factor IX. J Thromb Haemost 2004; 2: 108295.
  • 22
    van Helden PM, van Haren SD, Fijnvandraat K, van den Berg HM, Voorberg J. Factor VIII-specific B cell responses in haemophilia A patients with inhibitors. Haemophilia 2010; 16: 3543.
  • 23
    Delignat S, Repessé Y, Navarrete AM, Meslier Y, Gupta N, Christophe OD, Kaveri SV, Lacroix-Desmazes S. Immunoprotective effect of von Willebrand factor towards therapeutic factor VIII in experimental haemophilia A. Haemophilia 2012; 18: 24854.
  • 24
    Herczenik E, van Haren SD, Wroblewska A, Kaijen P, van den Biggelaar M, Meijer AB, Martinez-Pomares L, ten Brinke A, Voorberg J. Uptake of blood coagulation factor VIII by dendritic cells is mediated via its C1 domain. J Allergy Clin Immunol 2012; 129: 5019.e5.
  • 25
    Bovenschen N, van Dijk KW, Havekes LM, Mertens K, van Vlijmen BJ. Clearance of coagulation factor VIII in very low-density lipoprotein receptor knockout mice. Br J Haematol 2004; 126: 7225.
  • 26
    Bovenschen N, Mertens K, Hu L, Havekes LM, van Vlijmen BJ. LDL receptor cooperates with LDL receptor-related protein in regulating plasma levels of coagulation factor VIII in vivo. Blood 2005; 106: 90612.
  • 27
    Saenko EL, Yakhyaev AV, Mikhailenko I, Strickland DK, Sarafanov AG. Role of the low density lipoprotein-related protein receptor in mediation of factor VIII catabolism. J Biol Chem 1999; 274: 3768592.
  • 28
    Sarafanov AG, Ananyeva NM, Shima M, Saenko EL. Cell surface heparan sulfate proteoglycans participate in factor VIII catabolism mediated by low density lipoprotein receptor-related protein. J Biol Chem 2001; 276: 119709.
  • 29
    Sarafanov AG, Makogonenko EM, Pechik IV, Radtke KP, Khrenov AV, Ananyeva NM, Strickland DK, Saenko EL. Identification of coagulation factor VIII A2 domain residues forming the binding epitope for low-density lipoprotein receptor-related protein. Biochemistry 2006; 45: 182940.
  • 30
    Meems H, van den Biggelaar M, Rondaij M, van der Zwaan C, Mertens K, Meijer AB. C1 domain residues Lys 2092 and Phe 2093 are of major importance for the endocytic uptake of coagulation factor VIII. Int J Biochem Cell Biol 2011; 43: 111421.
  • 31
    Dasgupta S, Navarrete AM, Andre S, Wootla B, Delignat S, Repesse Y, Bayry J, Nicoletti A, Saenko EL, d’Oiron R, Jacquemin M, Saint-Remy JM, Kaveri SV, Lacroix-Desmazes S. Factor VIII bypasses CD91/LRP for endocytosis by dendritic cells leading to T-cell activation. Haematologica 2008; 93: 839.
  • 32
    Meems H, Meijer AB, Cullinan DB, Mertens K, Gilbert GE. Factor VIII C1 domain residues Lys 2092 and Phe 2093 contribute to membrane binding and cofactor activity. Blood 2009; 114: 393846.
  • 33
    Wroblewska A, van Haren SD, Herczenik E, Kaijen P, Ruminska A, Jin S-Y, Zheng XL, van den Biggelaar M, ten Brinke A, Meijer AB, Voorberg J. Modification of an exposed loop in the C1 domain reduces immune responses to factor VIII in hemophilia A mice. Blood 2012; 119: 5294300.
  • 34
    Behrmann M, Pasi J, Saint-Remy JM, Kotitschke R, Kloft M. von Willebrand factor modulates factor VIII immunogenicity: comparative study of different factor VIII concentrates in a haemophilia A mouse model. Thromb Haemost 2002; 88: 2219.
  • 35
    Delignat S, Dasgupta S, Andre S, Navarrete AM, Kaveri SV, Bayry J, Andre MH, Chtourou S, Tellier Z, Lacroix-Desmazes S. Comparison of the immunogenicity of different therapeutic preparations of human factor VIII in the murine model of hemophilia A. Haematologica 2007; 92: 14236.
  • 36
    Kallas A, Kuuse S, Maimets T, Pooga M. von Willebrand factor and transforming growth factor-beta modulate immune response against coagulation factor VIII in FVIII-deficient mice. Thromb Res 2007; 120: 91119.
  • 37
    Qadura M, Waters B, Burnett E, Chegeni R, Bradshaw S, Hough C, Othman M, Lillicrap D. Recombinant and plasma-derived factor VIII products induce distinct splenic cytokine microenvironments in hemophilia A mice. Blood 2009; 114: 87180.
  • 38
    Reipert BM, Schoppmann A, Schwarz HP. A caution on the use of murine hemophilia models for comparative immunogenicity studies of FVIII products with different protein compositions. Thromb Haemost 2003; 89: 111012.
  • 39
    Dasgupta S, Repesse Y, Bayry J, Navarrete AM, Wootla B, Delignat S, Irinopoulou T, Kamate C, Saint-Remy JM, Jacquemin M, Lenting PJ, Borel-Derlon A, Kaveri SV, Lacroix-Desmazes S. VWF protects FVIII from endocytosis by dendritic cells and subsequent presentation to immune effectors. Blood 2007; 109: 61012.
  • 40
    Goudemand J, Rothschild C, Demiguel V, Vinciguerrat C, Lambert T, Chambost H, Borel-Derlon A, Claeyssens S, Laurian Y, Calvez T. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. Blood 2006; 107: 4651.
  • 41
    Gouw SC, van der Bom JG, Auerswald G, Ettinghausen CE, Tedgard U, van den Berg HM. Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: the CANAL cohort study. Blood 2007; 109: 46937.
  • 42
    Vlot AJ, Koppelman SJ, van den Berg MH, Bouma BN, Sixma JJ. The affinity and stoichiometry of binding of human factor VIII to von Willebrand factor. Blood 1995; 85: 31507.
  • 43
    Mannucci PM, Gringeri A, Peyvandi F, Santagostino E. Factor VIII products and inhibitor development: the SIPPET study (survey of inhibitors in plasma-product exposed toddlers). Haemophilia 2007; 13: 658.
  • 44
    Gringeri A. VWF/FVIII concentrates in high-risk immunotolerance: the RESIST study. Haemophilia 2007; 5: 737.
  • 45
    Gaberc-Porekar V, Zore I, Podobnik B, Menart V. Obstacles and pitfalls in the PEGylation of therapeutic proteins. Curr Opin Drug Discov Devel 2008; 11: 24250.
  • 46
    Mei B, Pan C, Jiang H, Tjandra H, Strauss J, Chen Y, Liu T, Zhang X, Severs J, Newgren J, Chen J, Gu JM, Subramanyam B, Fournel MA, Pierce GF, Murphy JE. Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment. Blood 2010; 116: 2709.
  • 47
    Paz P, Xie J, Fuelle L, Shiroma D, Wu J, Liu P, Koellnberger M, Jacquemin M, Lavendhomme R, Laux V, Murphy J, Aswad F. PEGylated FVIII exhibits reduced immunogenicity in hemophilia A mice and in vitro in human cells. Haemophilia 2012; 18: PO-WE-125. Available at: http://www.journalth.com/about.php.
  • 48
    van Helden PM, Unterthurner S, Hermann C, Schuster M, Ahmad RU, Schiviz AN, Weiller M, Antoine G, Turecek PL, Muchitsch EM, Schwarz HP, Reipert BM. Maintenance and break of immune tolerance against human factor VIII in a new transgenic hemophilic mouse model. Blood 2011; 118: 3698707.
  • 49
    Peng A, Kosloski MP, Nakamura G, Ding H, Balu-Iyer SV. PEGylation of a factor VIII–phosphatidylinositol complex: pharmacokinetics and immunogenicity in hemophilia A mice. AAPS J 2012; 14: 3542.
  • 50
    Powell JS, Josephson NC, Quon D, Ragni MV, Cheng G, Li E, Jiang H, Li L, Dumont JA, Goyal J, Zhang X, Sommer J, McCue J, Barbetti M, Luk A, Pierce GF. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood 2012; 119: 30317.
  • 51
    Dumont JA, Liu T, Low SC, Zhang X, Kamphaus G, Sakorafas P, Fraley C, Drager D, Reidy T, McCue J, Franck HW, Merricks EP, Nichols TC, Bitonti AJ, Pierce GF, Jiang H. Prolonged activity of a recombinant factor VIII–Fc fusion protein in hemophilia A mice and dogs. Blood 2012; 119: 302430.
  • 52
    Akilesh S, Christianson GJ, Roopenian DC, Shaw AS. Neonatal FcR expression in bone marrow-derived cells functions to protect serum IgG from catabolism. J Immunol 2007; 179: 45808.
  • 53
    Lei TC, Scott DW. Induction of tolerance to factor VIII inhibitors by gene therapy with immunodominant A2 and C2 domains presented by B cells as Ig fusion proteins. Blood 2005; 105: 486570.
  • 54
    Liu T, Hoehn T, Patarroyo-White S, Pierce G, Jiang H. Evaluation of antibody responses to rFVIIIFc compared to Xyntha and Advate in hemophilia A mice. Haemophilia. 2012; 18: FP-WE-04.2-5. Available at: http://www.journalth.com/about.php.
  • 55
    Krishnamoorthy S, Valee S, Liu T, Drager D, Light D, Pierce G, Jiang H. Cell-mediated immune response to recombinant factor VIII–Fc in hemophilia A mice. Haemophilia 2012; 18: PO-WE-113, Available at: http://www.journalth.com/about.php.
  • 56
    Pfistershammer K, Stockl J, Siekmann J, Turecek PL, Schwarz HP, Reipert BM. Recombinant factor VIII and factor VIII–von Willebrand factor complex do not present danger signals for human dendritic cells. Thromb Haemost 2006; 96: 30916.
  • 57
    Esmon CT. Interactions between the innate immune and blood coagulation systems. Trends Immunol 2004; 25: 53642.
  • 58
    Ruf W. Emerging roles of tissue factor in viral hemorrhagic fever. Trends Immunol 2004; 25: 4614.
  • 59
    Bernard GR, Vincent J-L, Laterre P-F, LaRosa SP, Dhainaut J-F, Lopez-Rodriguez A, Steingrub JS, Garber GE, Helterbrand JD, Ely EW, Fisher CJ. Efficacy and safety of recombinant human activated protein C for severe sepsis. N Engl J Med 2001; 344: 699709.
  • 60
    Opal SM. The nexus between systemic inflammation and disordered coagulation in sepsis. J Endotoxin Res 2004; 10: 1259.
  • 61
    Kahn ML, Nakanishi-Matsui M, Shapiro MJ, Ishihara H, Coughlin SR. Protease-activated receptors 1 and 4 mediate activation of human platelets by thrombin. J Clin Invest 1999; 103: 87987.
  • 62
    van der Poll T, Levi M, Hack CE, ten Cate H, van Deventer SJ, Eerenberg AJ, de Groot ER, Jansen J, Gallati H, Büller HR. Elimination of interleukin 6 attenuates coagulation activation in experimental endotoxemia in chimpanzees. J Exp Med 1994; 179: 12539.
  • 63
    Riewald M, Kravchenko VV, Petrovan RJ, O’Brien PJ, Brass LF, Ulevitch RJ, Ruf W. Gene induction by coagulation factor Xa is mediated by activation of protease-activated receptor 1. Blood 2001; 97: 310916.
  • 64
    Niessen F, Schaffner F, Furlan-Freguia C, Pawlinski R, Bhattacharjee G, Chun J, Derian CK, Andrade-Gordon P, Rosen H, Ruf W. Dendritic cell PAR1–S1P3 signalling couples coagulation and inflammation. Nature 2008; 452: 6548.
  • 65
    Skupsky J, Zhang AH, Su Y, Scott DW. A role for thrombin in the initiation of the immune response to therapeutic factor VIII. Blood 2009; 114: 47418.
  • 66
    Sawamoto Y, Prescott R, Zhong D, Saenko EL, Mauser-Bunschoten E, Peerlinck K, van den Berg M, Scandella D. Dominant C2 domain epitope specificity of inhibitor antibodies elicited by a heat pasteurized product, factor VIII CPS-P, in previously treated hemophilia A patients without inhibitors. Thromb Haemost 1998; 79: 628.
  • 67
    Meeks SL, Cox CL, Healey JF, Parker ET, Doshi BS, Gangadharan B, Barrow RT, Lollar P. A major determinant of the immunogenicity of factor VIII in a murine model is independent of its procoagulant function. Blood 2012; 120: 251220.
  • 68
    Gouw SC, van den Berg HM, le Cessie S, van der Bom JG. Treatment characteristics and the risk of inhibitor development: a multicenter cohort study among previously untreated patients with severe hemophilia A. J Thromb Haemost 2007; 5: 138390.
  • 69
    Mancuso ME, Graca L, Auerswald G, Santagostino E. Haemophilia care in children – benefits of early prophylaxis for inhibitor prevention. Haemophilia 2009; 1: 814.
  • 70
    Jacquemin M, Vantomme V, Buhot C, Lavend’homme R, Burny W, Demotte N, Chaux P, Peerlinck K, Vermylen J, Maillere B, van der Bruggen P, Saint-Remy JM. CD4+ T-cell clones specific for wild-type factor VIII: a molecular mechanism responsible for a higher incidence of inhibitor formation in mild/moderate hemophilia A. Blood 2003; 101: 13518.
  • 71
    Reding MT, Okita DK, Diethelm-Okita BM, Anderson TA, Conti-Fine BM. Epitope repertoire of human CD4(+) T cells on the A3 domain of coagulation factor VIII. J Thromb Haemost 2004; 2: 138594.
  • 72
    Pratt KP, Qian J, Ellaban E, Okita DK, Diethelm-Okita BM, Conti-Fine B, Scott DW. Immunodominant T-cell epitopes in the factor VIII C2 domain are located within an inhibitory antibody binding site. Thromb Haemost 2004; 92: 5228.
  • 73
    Reipert BM, Steinitz KN, van Helden PM, Unterthurner S, Schuster M, Ahmad RU, Ilas J, Schwarz HP. Opportunities and limitations of mouse models humanized for HLA class II antigens. J Thromb Haemost 2009; 7(Suppl. 1): 927.
  • 74
    Steinitz KN, van Helden PM, Binder B, Wraith DC, Unterthurner S, Hermann C, Schuster M, Ahmad RU, Weiller M, Lubich C, de la Rosa M, Schwarz HP, Reipert BM. CD4+ T-cell epitopes associated with antibody responses after intravenously and subcutaneously applied human FVIII in humanized hemophilic E17 HLA-DRB1*1501 mice. Blood 2012; 119: 407382.
  • 75
    Gregersen JW, Holmes S, Fugger L. Humanized animal models for autoimmune diseases. Tissue Antigens 2004; 63: 38394.
  • 76
    Mangalam AK, Rajagopalan G, Taneja V, David CS. HLA class II transgenic mice mimic human inflammatory diseases. Adv Immunol 2008; 97: 65147.
  • 77
    Hay CR, Ollier W, Pepper L, Cumming A, Keeney S, Goodeve AC, Colvin BT, Hill FG, Preston FE, Peake IR. HLA class II profile: a weak determinant of factor VIII inhibitor development in severe haemophilia A. UKHCDO Inhibitor Working Party. Thromb Haemost 1997; 77: 2347.
  • 78
    Moise L, Song C, Martin WD, Tassone R, De Groot AS, Scott DW. Effect of HLA DR epitope de-immunization of factor VIII in vitro and in vivo. Clin Immunol 2012; 142: 32031.
  • 79
    Reding MT, Okita DK, Diethelm-Okita BM, Anderson TA, Conti-Fine BM. Human CD4+ T-cell epitope repertoire on the C2 domain of coagulation factor VIII. J Thromb Haemost 2003; 1: 177784.
  • 80
    Ettinger RA, James EA, Kwok WW, Thompson AR, Pratt KP. Lineages of human T-cell clones, including T helper 17/T helper 1 cells, isolated at different stages of anti-factor VIII immune responses. Blood 2009; 114: 14238.
  • 81
    Ettinger RA, James EA, Kwok WW, Thompson AR, Pratt KP. HLA-DR-restricted T-cell responses to factor VIII epitopes in a mild haemophilia A family with missense substitution A2201P. Haemophilia 2010; 16: 4455.
  • 82
    Hay CR. Factor VIII inhibitors in mild and moderate-severity haemophilia A. Haemophilia 1998; 4: 55863.
  • 83
    Gouw SC, van den Berg HM, Oldenburg J, Astermark J, de Groot PG, Margaglione M, Thompson AR, van Heerde W, Boekhorst J, Miller CH, le Cessie S, van der Bom JG. F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis. Blood 2012; 119: 292234.
  • 84
    Viel KR, Ameri A, Abshire TC, Iyer RV, Watts RG, Lutcher C, Channell C, Cole SA, Fernstrom KM, Nakaya S, Kasper CK, Thompson AR, Almasy L, Howard TE. Inhibitors of factor VIII in black patients with hemophilia. N Engl J Med 2009; 360: 161827.
  • 85
    Gunasekera D, Ettinger RA, Hughes RJ, Epstein MS, Barrett JC, Thompson AA, Withycombe J, Pratt KP. Potential immunogenicity of amino acid sequences encoded by Ns-SNPs in factor VIII. ASH Annual Meeting Abstracts 2011; 118: 3329.
  • 86
    Liu MER, James E, Lewis K, Pratt K. Identification of potential T-cell epitopes in factor VIII using peptide microarrays. Haemophilia. 2012; 18: PO-WE-132, Available at http://www.journalth.com/about.php.