A role for von Willebrand factor in immune tolerance induction in patients with haemophilia A and inhibitors?

Blood Transfusion

Volumn 9, Issue SUPPL. 2, 2011, Pages

  Di Minno, Giovanni ^a   Coppola, Antonio ^a  

^a FEDERICO II UNIVERSITY HOSPITAL   (Italy)

Author keywords

Factor VIII concentrates; Haemophilia A; Immune tolerance induction; Inhibitors; Von Willebrand factor

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 CONCENTRATE; INTERLEUKIN 10; RECOMBINANT BLOOD CLOTTING FACTOR 8; TRANSFORMING GROWTH FACTOR BETA; VON WILLEBRAND FACTOR;

ANTIGEN PRESENTATION; ANTIGEN PRESENTING CELL; BINDING AFFINITY; CLINICAL FEATURE; DENDRITIC CELL; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG HYDROLYSIS; DRUG MEGADOSE; DRUG TOLERABILITY; ENDOCYTOSIS; GENE EXPRESSION; HEMOPHILIA A; HUMAN; IMMUNE RESPONSE; IMMUNE SYSTEM; IMMUNE TOLERANCE INDUCTION; IMMUNOGENICITY; IMMUNOLOGICAL TOLERANCE; IN VITRO STUDY; LOW DRUG DOSE; NONHUMAN; PROGNOSIS; REVIEW; UPREGULATION;

ANIMALS; AUTOANTIBODIES; HEMOPHILIA A; HUMANS; IMMUNE TOLERANCE; TREATMENT OUTCOME; VON WILLEBRAND FACTOR;

EID: 79959806956     PISSN: 17232007     EISSN: None     Source Type: Journal    
DOI: 10.2450/2011.004S     Document Type: Review

References (52)

1. Astermark J, Santagostino E, Keith Hoots W. Clinical issues in inhibitors. Haemophilia 2010; 16(Suppl 5): 54-60.
2. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A. A systematic review. Haemophilia 2003; 9: 418-35.
3. Gringeri A, Mantovani LG, Scalone L, Mannucci PM. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood 2003; 102: 2358-63. (Pubitemid 37193570)
4. UK Haemophilia Centre Doctors' Organization. The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977-99. J Thromb Haemost 2004; 2: 1047-54.
5. Scalone L, Mantovani LG, Mannucci PM, Gringeri A. Quality of life is associated to the orthopaedic status in haemophilic patients with inhibitors. Haemophilia 2006; 12: 154-62.
6. Morfini M, Haya S, Tagariello G, et al. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia 2007; 13: 606-12. (Pubitemid 47338004)
7. Hay CR, Baglin TP, Collins PW, et al. The diagnosis and management of factor VIII and IX inhibitors: a guideline from the UK Haemophilia Centre Doctors Organization (UKHCDO). Br J Haematol 2006; 133: 591-605. (Pubitemid 43742773)
8. DiMichele DM, Hoots WK, Pipe SW, et al. International workshop on immune tolerance induction: consensus recommendations. Haemophilia 2007; 13(Suppl. 1): 1-22. (Pubitemid 47009242)
9. Coppola A, Santoro C, Tagliaferri A, et al. Understanding inhibitor development in haemophilia A: towards clinical prediction and prevention strategies. Haemophilia 2010; 16(Suppl 1): 13-9.
10. Waters B, Lillicrap D. The molecular mechanisms of immunomodulation and tolerance induction to factor VIII. J Thromb Haemost 2009; 7: 1446-56.
11. Coppola A, Di Minno MND, Santagostino E. Optimizing management of immune tolerance induction in patients with severe haemophilia A and inhibitors: towards evidence-based approaches. Br J Haematol 2010; 150: 515-28.
12. Mannucci PM. Plasma-derived versus recombinant factor VIII concentrates for the treatment of haemophilia A: plasma-derived is better. Blood Transfusion 2010; 8: 288-91.
13. Franchini M. Plasma-derived versus recombinant factor VIII concentrates for the treatment of haemophilia A: recombinant is better. Blood Transfusion 2010; 8: 292-6.
14. Hay CRM, Goldberg I, Foulkes M, DiMichele DM, on behalf of the International Immune Tolerance Study Group. International prospective randomized immune tolerance (ITI) study: interim analysis of therapeutic efficacy and safety. Haemophilia 2010; 16: 405 (abs P5).
15. Gringeri A. VWF/FVIII concentrates in high-risk immunotolerance: the RESIST study. Haemophilia. 2007; 13(Suppl. 5): 73-7. (Pubitemid 350253409)
16. Kreuz W, Escuriola-Ettingshausen C, Auerswald G, et al. Immune tolerance induction (ITI) in haemophilia A with inhibitors: the choice of concentrate affecting success. Haematologica 2001; 86(Suppl. 4): 16-20.
17. Auerswald G, Spranger T, Brackmann HH. The role of plasma-derived factor VIII/von Willebrand concentrates in the treatment of hemophilia A patients. Haematologica 2003; 88(Suppl. 9): 21-5.
18. Mariani G, Kroner B, for the Immune Tolerance Study Group. Immune tolerance in hemophilia with inhibitors: predictors of success. Haematologica. 2001; 86: 1186-93.
19. Lenk H. The German registry of immune tolerance treatment in hemophilia - 1999 update. Haematologica 2000; 85: 45-7.
20. Haya S, Lopez MF, Aznar JA, Batlle J. Immune tolerance treatment in haemophilia patients with inhibitors: the Spanish Registry. Haemophilia 2001; 7: 154-9. (Pubitemid 32243797)
21. DiMichele DM, Kroner BL. The North American Immune Tolerance Registry: practices, outcomes, outcome predictors. Thromb Haemost. 2002; 87: 52-7. (Pubitemid 34092575)
22. Coppola A, Margaglione M, Santagostino E, et al. Factor VIII gene (F8) mutations as predictors of outcome in immune tolerance induction of patients with severe hemophilia A and high-responding inhibitors. J Thromb Haemost 2009; 7: 1809-15.
23. Orsini F, Rotschild C, Beurrier P, et al. Immune tolerance induction with highly purified plasma-derived factor VIII containing von Willebrand factor in hemophilia A patients with high-responding inhibitors. Haematologica 2005; 90: 1288-90. (Pubitemid 41375296)
24. Gringeri A, Musso R, Mazzucconi MG, et al for the RITS-FITNHES Study Group. Immune tolerance induction with a high-purity von Willebrand factor/VIII complex concentrate in haemophilia A patients with inhibitors at high risk of a poor response. Haemophilia 2007; 13: 373-9.
25. Grenninger DA, Saint-Remy JM, Jacquemin M, et al. The use of factor VIII/von Willebrand factor concentrate for immune tolerance induction in haemophilia A patients with high-titre inhibitors: association of clinical outcome with inhibitor epitope profile. Haemophilia 2008; 14: 295-302. (Pubitemid 351338690)
26. Kurth MA, DiMichele D, Sexauer C, et al. Immune tolerance therapy utilizing factor VIII/von Willebrand factor concentrate in haemophilia A patients with high titre factor VIII inhibitors. Haemophilia 2008; 14: 50-5.
27. Batlle J, Lopez MF, Brackmann HH, et al. Induction of immune tolerance with recombinant factor VIII in haemophilia A patients with inhibitors. Haemophilia 1999; 5: 431-5. (Pubitemid 30003772)
28. Rothschild C, Laurian Y, Satre EP, et al. French previously untreated patients with severe hemophilia A after exposure to recombinant Factor VIII: incidence of inhibitor and evaluation of immune tolerance. Thromb Haemost 1998; 80: 779-83. (Pubitemid 28521722)
29. Smith MP, Spence KJ, Waters EL, et al. Immune tolerance therapy for haemophilia A patients with acquired factor VIII alloantibodies: comprehensive analysis of experience at a single institution. Thromb Haemost 1999; 81: 35-8. (Pubitemid 29041646)
30. Barnes C, Rivard GE, Poon M-C, et al. Canadian multiinstitutional survey of immune tolerance therapy (ITT) - experience with the use of recombinant factor VIII for ITT. Haemophilia 2006; 12: 1-6.
31. Rocino A, Santagostino E, Mancuso ME, Mannucci PM. Immune tolerance induction with recombinant factor VIII in hemophilia A patients with high responding inhibitors. Haematologica 2006; 91: 558-61.
32. Valentino LA, Recht M, Dipaola J, et al. Experience with a third generation recombinant factor VIII concentrate (AdvateÆÊ) for immune tolerance induction in patients with haemophilia A. Haemophilia 2009; 15: 727-32.
33. Coppola A, Arbasi MC, Biasoli C, et al. Immune tolerance induction (ITI) with a FVIII concentrate with high VWF content in inhibitor patients with previous unsuccessful ITI and/or negative predictors of ITI response. Haemophilia 2010; 16(Suppl 6): 70-1 (abs 17P23).
34. Kreuz W. The role of VWF for the success of immune tolerance induction. Thromb Res 2008; 122(Suppl 2): S7-S12. (Pubitemid 351783318)
35. Suzuki T, Arai M, Amano K, et al. FVIII inhibitor antibodies with C2 domain specificity are less inhibitory to FVIII complexed with VWF. Thromb Haemost 1996; 76: 749-54. (Pubitemid 26388181)
36. Gensana M, Altisent C, Aznar JA, et al. Influence of VWF on the reactivity of human FVIII inhibitors with FVIII. Haemophilia 2001; 7: 369-74. (Pubitemid 32677655)
37. Kallas A, Talpsep T. Von Willebrand factor in factor VIII concentrates protects against neutralization by factor VIII antibodies of haemophilia A patients. Haemophilia 2001; 7: 375-80. (Pubitemid 32677656)
38. Astermark J, Voorberg J, Lenk H, et al. Impact of inhibitor epitope profile on the neutralizing effect against plasma-derived and recombinant factor VIII concentrates in vitro. Haemophilia 2003; 9: 567-72. (Pubitemid 37236091)
39. Tagariello G, Zanotto D, Radossi P, et al. In vitro reactivity of FVIII inhibitors with von Willebrand factor in different commercial FVIII concentrates. Am J Hematol 2007: 82: 460-2.
40. Salvagno GL, Astermark J, Ekman M, et al. Impact of different inhibitor reactivities with commercial FVIII concentrates on thrombin generation. Haemophilia 2007; 13: 51-6. (Pubitemid 46002345)
41. Franchini M, Lippi G. Von Willebrand factorcontaining factor VIII concentrates and inhibitors in haemophilia A. A critical literature review. Thromb Haemost 2010; 104; 931-40.
42. Prescott R, Nakai H, Saenko EL, et al. The inhibitor antibody response is more complex in hemophilia A than in most nonhemophiliacs with factor VIII autoantibodies. Blood 1997; 89: 3663-71. (Pubitemid 27227089)
43. Lacroix Desmazes S, Bayry J, Misra N, et al. The prevalence of proteolytic antibodies against factor VIII in hemophilia A. N Engl J Med 2002; 346; 662-7. (Pubitemid 34815870)
44. Dasgupta S, Repesse Y, Bayry J, et al. VWF protects from endocytosis by dendritic cells and subsequent presentation to immune effectors. Blood 2007; 109: 610-2. (Pubitemid 46105959)
45. Delignat S, Dasgupta S, André S, et al. Comparison of the immunogenicity of different therapeutic preparations of human factor VIII in the murine model of hemophilia A. Haematologica 2007; 92: 1423-6. (Pubitemid 350144163)
46. Qadura M, Waters B, Burnett E, et al. Recombinant and plasma-derived factor VIII products induce distinct splenic cytokine environment in hemophilia A mice. Blood 2009; 114: 871-80.
47. Vlot AJ, Koppelman SJ, Meijers JC, et al. Kinetics of factor VIII-von Willebrand factor association. Blood 1996; 87: 1809-16. (Pubitemid 26074100)
48. Lin Y, Yang X, Chevrier MC, et al. Relationships between factor VIII:Ag and factor VIII in recombinant and plasma-derived factor VIII concentrates. Haemophilia 2004; 10: 459-69. (Pubitemid 39317754)
49. Hodge G, Han P. Effect of intermediate-purity factor VIII (FVIII) concentrate on lymphocyte proliferation and apoptosis: transforming growth factor-beta is a significant immunomodulatory component of FVIII. Br J Haematol 2001; 115, 376-81. (Pubitemid 34203506)
50. Hausl C, Ahmad RU, Schwarz HP, et al. Preventing restimulation of memory B cells in hemophilia A: a potential new strategy for the treatment of antibody-dependent immune disorders. Blood 2004; 104: 115-22. (Pubitemid 38879846)
51. Viel KR, Ameri A, Abshire TC, et al. Inhibitors of factor VIII in black patients with hemophilia. N Engl J Med 2009; 360: 1618-27.
52. van Helden PM, Kaijen PH, Mauser-Bunschoten EP, et al. Domain specificity of factor VIII inhibitors during immune tolerance induction in patients with haemophilia A. Haemophilia 2010; 16; 892-901.