Page 39 - 2019_02-Haematologica-web

Immune response to FVIII in hemophilia patients
sense of self. Science. 2002;296(5566):301-
305.
16. Navarrete A, Dasgupta S, Delignat S, et al.
Splenic marginal zone antigen-presenting cells are critical for the primary allo-immune response to therapeutic factor VIII in hemo- philia A. J Thromb Haemost. 2009;7(11): 1816-1823.
17. Lai JD, Cartier D, Hartholt RB, et al. Early cellular interactions and immune transcrip- tome profiles in human factor VIII-exposed hemophilia A mice. J Thromb Haemost. 2018;16(3):533-545.
18. Dasgupta S, Navarrete AM, Bayry J, et al. A role for exposed mannosylations in presen- tation of human therapeutic self-proteins to CD4+ T lymphocytes. Proc Natl Acad Sci U S A. 2007;104(21):8965-8970.
19. Herczenik E, van Haren SD, Wroblewska A, et al. Uptake of blood coagulation factor VIII by dendritic cells is mediated via its C1 domain. J Allergy Clin Immunol. 2012;129 (2):501-509.
20. Janeway CA, Jr. Approaching the asymp- tote? Evolution and revolution in immunol- ogy. Cold Spring Harb Symp Quant Biol. 1989;54 (Pt 1):1-13.
21. Matzinger P. Tolerance, danger, and the extended family. Annu Rev Immunol. 1994;12:991-1045.
22. Astermark J, Altisent C, Batorova A, et al. Non-genetic risk factors and the develop- ment of inhibitors in haemophilia: a com- prehensive review and consensus report. Haemophilia. 2010;16(5):747-766.
23. Reipert BM, Ahmad RU, Turecek PL, et al. Characterization of antibodies induced by human factor VIII in a murine knockout model of hemophilia A. Thromb Haemost. 2000;84(5):826-832.
24. Gouw SC, van den Berg HM, Fischer K, et al. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013;121(20):4046-4055.
25. Dasgupta S, Repesse Y, Bayry J, et al. VWF protects FVIII from endocytosis by dendritic cells and subsequent presentation to immune effectors. Blood. 2007;109(2):610- 612.
26. Pradeu T, Jaeger S, Vivier E. The speed of change: towards a discontinuity theory of immunity? Nat Rev Immunol. 2013;13(10): 764-769.
27. Ing M, Gupta N, Teyssandier M, et al. Immunogenicity of long-lasting recombi- nant factor VIII products. Cell Immunol. 2016;301:40-48.
28. Teyssandier M, Andre S, Gupta N, et al. Therapeutic factor VIII does not trigger TLR1.2 and TLR2.6 signalling in vitro. Haemophilia. 2013;19(3):399-402.
29. 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(3):309-316.
30. Cirino G, Cicala C, Bucci MR, Sorrentino L, Maraganore JM, Stone SR. Thrombin func- tions as an inflammatory mediator through activation of its receptor. J Exp Med. 1996;183(3):821-827.
31. 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(21):4741-4748.
32. Gangadharan B, Delignat S, Ollivier V, et al. Role of coagulation-associated processes on factor VIII immunogenicity in a mouse model of severe hemophilia A. J Thromb
Haemost. 2014;12(12):2065–2069.
33. Lai JD, Moorehead PC, Sponagle K, et al. Concurrent influenza vaccination reduces anti-FVIII antibody responses in murine hemophilia A. Blood. 2016;127(26):3439-
3449.
34. Platokouki H, Fischer K, Gouw SC, et al.
Vaccinations are not associated with inhibitor development in boys with severe haemophilia A. Haemophilia. 2018;24(2): 283-290.
35. Dimitrov JD, Dasgupta S, Navarrete AM, et al. Induction of heme oxygenase-1 in factor VIII-deficient mice reduces the immune response to therapeutic factor VIII. Blood. 2010;115(13):2682-2685.
36. Peyron I, Dimitrov JD, Delignat S, et al. Haemarthrosis and arthropathy do not favour the development of factor VIII inhibitors in severe haemophilia A mice. Haemophilia. 2015;21(1):e94-98.
37. Kurnik K, Bidlingmaier C, Engl W, Chehadeh H, Reipert B, Auerswald G. New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor development. Haemophilia. 2009;16(2):256-262.
38. Auerswald G, Bidlingmaier C, Kurnik K. Early prophylaxis/FVIII tolerization regimen that avoids immunological danger signals is still effective in minimizing FVIII inhibitor developments in previously untreated patients--long-term follow-up and continu- ing experience. Haemophilia. 2012;18(1): e18-20.
39. Bexborn F, Andersson PO, Chen H, Nilsson B, Ekdahl KN. The tick-over theory revisit- ed: formation and regulation of the soluble alternative complement C3 convertase (C3(H2O)Bb). Mol Immunol. 2008;45(8): 2370-2379.
40. Zewde N, Gorham RD, Jr., Dorado A, Morikis D. Quantitative modeling of the alternative pathway of the complement sys- tem. PLoS One. 2016;11(3):e0152337.
41. Rayes J, Ing M, Delignat S, et al. Complement C3 is a novel modulator of the anti-factor VIII immune response. Haematologica. 2018;103(2):351-360.
42. Lacroix-Desmazes S, Navarrete AM, Andre S, Bayry J, Kaveri SV, Dasgupta S. Dynamics of factor VIII interactions determine its immunologic fate in hemophilia A. Blood. 2008;112(2):240-249.
43. Sakaguchi S, Vignali DA, Rudensky AY, Niec RE, Waldmann H. The plasticity and stabili- ty of regulatory T cells. Nat Rev Immunol. 2013;13(6):461-467.
44. Wang X, Terhorst C, Herzog RW. In vivo induction of regulatory T cells for immune tolerance in hemophilia. Cell Immunol. 2016;301:18-29.
45. Hu GL, Okita DK, Diethelm-Okita BM, Conti-Fine BM. Recognition of coagulation factor VIII by CD4+ T cells of healthy humans. J Thromb Haemost. 2003;1(10): 2159-2166.
46. Reding M, Wu H, Krampf M, et al. Sensitization of CD4+ T cells to coagulation factor VIII: response in congenital and acquired hemophilia patients and in healthy subjects. Thromb Haemost. 2000;84(4):643- 652.
47. Meunier S, Menier C, Marcon E, Lacroix- Desmazes S, Maillere B. CD4 T cells specific for factor VIII are present at high frequency in healthy donors and comprise naïve and memory cells. blood advances. 2017;1(21):1842-1847.
48. Delluc S, Ravot G, Maillere B. Quantitative analysis of the CD4 T-cell repertoire specific
to therapeutic antibodies in healthy donors.
FASEB J. 2011;25(6):2040-2048.
49. Delluc S, Ravot G, Maillere B.
Quantification of the preexisting CD4 T-cell repertoire specific for human erythropoietin reveals its immunogenicity potential. Blood. 2010;116(22):4542-4545.
50. Hamze M, Meunier S, Karle A, et al. Characterization of CD4 T cell epitopes of infliximab and rituximab identified from healthy donors. Front Immunol. 2017;8:500.
51. Pinto S, Michel C, Schmidt-Glenewinkel H, et al. Overlapping gene coexpression pat- terns in human medullary thymic epithelial cells generate self-antigen diversity. Proc Natl Acad Sci USA. 2013;110(37):E3497- 3505.
52. Kamate C, Lenting PJ, van den Berg HM, Mutis T. Depletion of CD4+/CD25high reg- ulatory T cells may enhance or uncover fac- tor VIII-specific T-cell responses in healthy individuals. J Thromb Haemost. 2007;5(3):611-613.
53. Algiman M, Dietrich G, Nydegger U, Boieldieu D, Sultan Y, Kazatchkine M. Natural antibodies to factor VIII (anti-hemo- philic factor) in healthy individuals. Proc Natl Acad Sci USA. 1992;89(9):3795-3799.
54. Whelan SF, Hofbauer CJ, Horling FM, et al. Distinct characteristics of antibody respons- es against factor VIII in healthy individuals and in different cohorts of hemophilia A patients. Blood. 2013;121(6):1039-1048.
55. Hofbauer CJ, Whelan SF, Hirschler M, et al. Affinity of FVIII-specific antibodies reveals major differences between neutralizing and nonneutralizing antibodies in humans. Blood. 2015;125(7):1180-1188.
56. Gilles JG, Saint-Remy JM. Healthy subjects produce both anti-factor VIII and specific anti-idiotypic antibodies. J Clin Invest. 1994;94(4):1496-1505.
57. Sakurai Y, Shima M, Tanaka I, Fukuda K, Yoshida K, Yoshioka A. Association of anti- idiotypic antibodies with immune tolerance induction for the treatment of hemophilia A with inhibitors. Haematologica. 2004;89(6): 696-703.
58. Dietrich G, Algiman M, Sultan Y, Nydegger UE, Kazatchkine MD. Origin of anti-idio- typic activity against anti-factor VIII autoan- tibodies in pools of normal human immunoglobulin G (IVIg). Blood. 1992;79 (11):2946-2951.
59. Moreau A, Lacroix-Desmazes S, Stieltjes N, et al. Antibodies to the FVIII light chain that neutralize FVIII procoagulant activity, are present in the plasma of non-responder patients with severe hemophilia A and healthy blood donors. Blood. 2000;95(11): 3435-3441.
60. Rossi F, Sultan Y, Kazatchkine MD. Anti- idiotypes against autoantibodies and alloan- tibodies to Factor VIII:C (anti-haemophilic factor) are present in therapeutic polyspecif- ic normal immunoglobulins. Clin Exp Immunol. 1988;74(2):311-316.
61. Eckhardt CL, van Velzen AS, Peters M, et al. Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemo- philia A. Blood. 2013;122(11):1954-1962.
62. Malhotra JD, Miao H, Zhang K, et al. Antioxidants reduce endoplasmic reticulum stress and improve protein secretion. Proc Natl Acad Sci U S A. 2008;105(47):18525- 18530.
63. Jacquemin M, Vantomme V, Buhot C, et al. CD4+ T-cell clones specific for wild-type factor VIII: a molecular mechanism respon- sible for a higher incidence of inhibitor for- mation in mild/moderate hemophilia A.
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