179
Views
4
CrossRef citations to date
0
Altmetric
Reviews

Immune tolerance induction for the eradication of inhibitors in patients with hemophilia A

, MD, , MD & , MD
Pages 1885-1896 | Published online: 06 Nov 2008

Bibliography

  • Pergantou H, Matsinos G, Papadopoulos A, et al. Comparative study of validity of clinical, X-ray and magnetic resonance imaging scores in evaluation and management of haemophilic arthropathy in children. Haemophilia 2006;12:241-7
  • CDC: Bleeding and clotting disorders surveillance. Summary report of UDC activity. Available from: http://www2a.cdc.gov/ncbddd/htcweb/UDC_Report/UDC_view1.asp?para1= NATION&para2=TREATH&para3= &ScreenWidth=1920&ScreenHeight=1200
  • White GC, Rosendaal F, Aledort LM, et al. Definitions in hemophilia: recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost 2001;85:560
  • Srivastava A, Giangrande P, Poon MC, et al. Guidelines for the management of hemophilia, 2006. Available from: http://www.wfh.org/index.asp?lang=EN
  • MASAC: Medical and Scientific Advisory Council Recommendation Concerning Prophylaxis (Regular Administration of Clotting Factor Concentrate to Prevent Bleeding). MASAC Recommendation 170. National Hemophilia Foundation 2006. Available from: http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=57&contentid=1007 [last accessed 12 August 2008]
  • Manco-Johnson M. Comparing prophylaxis with episodic treatment in haemophilia A: implications for clinical practice. Haemophilia 2007;13(Suppl 2):4-9
  • Brackmann HH, Wallny T. Immune tolerance: high-dose regimen. In: Rodriguez-Merchan EC, Lee CA, editors. Inhibitors in patients with hemophilia. Oxford: Blackwell; 2002. p. 45-8
  • Hay CR, Ludlam CA, Colvin BT, et al. Factor VIII inhibitors in mild and moderate-severity haemophilia A. UK Haemophilia Centre Directors Organisation. Thromb Haemost 1998;79:762-6
  • Aledort L. Inhibitors in hemophilia patients: current status and management. Am J Hematol 1994;47:208-17
  • Bray GL, Gomperts ED, Courter S, et al. A multicenter study of recombinant factor VIII (Recombinate): safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. The Recombinate Study Group. Blood 1994;83:2428-35
  • Gouw SC, van den Berg HM, le Cessie S, et al. Treatment characteristics and the risk of inhibitor development: a multi-center cohort study among previously untreated patients with severe hemophilia A. J Thromb Haemost 2007;5:1383-90
  • Kasper CK. Diagnosis and management of inhibitors to factors VIII and IX. An introductory discussion for physicians. Treatment of Hemophilia. World Federation of Hemophilia; September 2004, no. 34
  • James EA, Kwok WW, Ettinger RA, et al. 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;5:2399-407
  • Ananyeva NM, Lacroix-Desmazes S, Hauser CA, et al. Inhibitors in hemophilia A: mechanisms of inhibition, management and perspectives. Blood Coagul Fibrinolysis 2004;15:109-24
  • Lacroix-Desmazes S, Moreau A, Sooryanarayana, et al. Catalytic activity of antibodies against factor VIII in patients with hemophilia A. Nat Med 1999;5:1044-7
  • Gilles JG, Arnout J, Vermylen J, et al. Anti-factor VIII antibodies of hemophiliac patients are frequently directed towards nonfunctional determinants and do not exhibit isotypic restriction. Blood 1993;82:2452-61
  • Dazzi F, Tison T, Vianello F, et al. High incidence of anti-FVIII antibodies against non-coagulant epitopes in haemophilia A patients: a possible role for the half-life of transfused FVIII. Br J Haematol 1996;93:688-93
  • Guery JC, Adorini L. Dendritic cells are the most efficient in presenting endogenous naturally processed self-epitopes to class II-restricted T cells. J Immunol 1995;154:536-44
  • Chesnut RW, Grey HM. Antigen presenting cells and mechanisms of antigen presentation. Crit Rev Immunol 1985;5:263-316
  • Bhardwaj N. Processing and presentation of antigens by dendritic cells: implications for vaccines. Trends Mol Med 2001;7:388-94
  • Dasgupta S, Repesse Y, Bayry J, et al. VWF protects FVIII from endocytosis by dendritic cells and subsequent presentation to immune effectors. Blood 2007;109:610-2
  • Lacroix-Desmazes S, Navarrete AM, Andre S, et al. Dynamics of factor VIII interactions determine its immunological fate in hemophilia A. Blood 2008;112:240-9
  • Kaveri SV, Dasgupta S, Andre S, et al. Factor VIII inhibitors: role of von Willebrand factor on the uptake of factor VIII by dendritic cells. Haemophilia 2007;13(Suppl 5):61-4
  • Reipert BM, van Helden PM, Schwarz HP, et al. Mechanisms of action of immune tolerance induction against factor VIII in patients with congenital haemophilia A and factor VIII inhibitors. Br J Haematol 2007;136:12-25
  • Chiller JM, Habicht GS, Weigle WO. Cellular sites of immunologic unresponsiveness. Proc Natl Acad Sci USA 1970;65:551-6
  • Rajewsky K. Clonal selection and learning in the antibody system. Nature 1996;381:751-8
  • Vinuesa CG, Tangye SG, Moser B, et al. Follicular B helper T cells in antibody responses and autoimmunity. Nat Rev Immunol 2005;5:853-65
  • Reding MT, Wu H, Krampf M, et al. CD4+ T cell response to factor VIII in hemophilia A, acquired hemophilia, and healthy subjects. Thromb Haemost 1999;82:509-15
  • Dasgupta S, Navarrete AM, Bayry J, et al. A role for exposed mannosylations in presentation of human therapeutic self-proteins to CD4+ T lymphocytes. Proc Natl Acad Sci USA 2007;104:8965-70
  • Astermark J, Berntorp E, White GC, et al. The Malmö International Brother Study (MIBS): further support for genetic predisposition to inhibitor development in hemophilia patients. Haemophilia 2001;7:267-72
  • Schwaab R, Brackmann HH, Meyer C, et al. Haemophilia A: mutation type determines risk of inhibitor formation. Thromb Haemost 1995,74:1402-6
  • Tuddenham EG, McVey JH. The genetic basis of inhibitor development in haemophilia A. Haemophilia 1998;4:543-5
  • Fakharzadeh SS, Kazazian HH Jr. Correlation between factor VIII genotype and inhibitor development in hemophilia A. Semin Thromb Hemost 2000;26:167-71
  • Oldenburg J, Schroder J, Brackmann HH, et al. Environmental and genetic factors influencing inhibitor development. Semin Hematol 2004;41:82-8
  • Gill JC. The role of genetics in inhibitor formation. Thromb Haemost 1999;82:500-4
  • Astermark J, Oldenburg J, Carlson J, et al. Polymorphisms in the TNFA gene and the risk of inhibitor development in patients with hemophilia A. Blood 2006;108:3739-45
  • Astermark J, Oldenburg J, Pavlova A, et al. Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A. Blood 2006;107:3167-72
  • Astermark J, Wang X, Oldenburg J, et al. Polymorphisms in the CTLA-4 gene and inhibitor development in patients with severe hemophilia A. J Thromb Haemost 2007;5:263-5
  • Aledort LM, DiMichele DM. Inhibitors occur more frequently in African-American and Latino haemophiliacs. Haemophilia 1998;4:68
  • Lusher J, Abildgaard C, Arkin S, et al. Human recombinant DNA-derived antihemophilic factor in the treatment of previously untreated patients with hemophilia A: final report on a hallmark clinical investigation. J Thromb Haemost 2004;2:574-83
  • Astermark J. Overview of inhibitors. Semin Hematol 2006;43:S3-7
  • Morado M, Villar A, Jimenez Yuste V, et al. Prophylactic treatment effects on inhibitor risk: experience in one centre. Haemophilia 2005;11:79-83
  • Santagostino E, Mancuso ME, Rocino A, et al. Environmental risk factors for inhibitor development in children with haemophilia A: a case-control study. Br J Haematol 2005;130:422-7
  • Gouw SC, Van Der Bom JG, Marijke van den Berg H. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood 2007;109:4648-54
  • Leissinger CA. Prevention of bleeds in hemophilia patients with inhibitors: emerging data and clinical direction. Am J Hematol 2004;77:187-93
  • Arkin S, Blei F, Fetten J, et al. Human coagulation factor FVIIa (recombinant) in the management of limb-threatening bleeds unresponsive to alternative therapies: results from the NovoSeven emergency-use programme in patients with severe haemophilia or with acquired inhibitors. Blood Coagul Fibrinolysis 2000;11:255-9
  • Hilgartner M, Aledort L, Andes A, et al. Efficacy and safety of vapor-heated anti-inhibitor coagulant complex in hemophilia patients. FEIBA Study Group. Transfusion 1990;30:626-30
  • Key NS, Aledort LM, Beardsley D, et al. Home treatment of mild to moderate bleeding episodes using recombinant factor VIIa (Novoseven) in haemophiliacs with inhibitors. Thromb Haemost 1998;80:912-8
  • Negrier C, Goudemand J, Sultan Y, et al. Multicenter retrospective study on the utilization of FEIBA in France in patients with factor VIII and factor IX inhibitors. French FEIBA Study Group. Factor Eight Bypassing Activity. Thromb Haemost 1997;77:1113-9
  • Shapiro AD, Gilchrist GS, Hoots WK, et al. Prospective, randomised trial of two doses of rFVIIa (NovoSeven) in haemophilia patients with inhibitors undergoing surgery. Thromb Haemost 1998;80:773-8
  • Tjonnfjord GE. Activated prothrombin complex concentrate (FEIBA) treatment during surgery in patients with inhibitors to FVIII/IX: the updated Norwegian experience. Haemophilia 2004;10(Suppl 2):41-5
  • Astermark J, Donfield SM, DiMichele DM, et al. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study. Blood 2007;109:546-51
  • Brackmann HH, Schwaab R, Effenberger W, et al. Antibodies to factor VIII in hemophilia A patients. Vox Sang 2000;78(Suppl 2):187-90
  • Leissinger CA. Use of prothrombin complex concentrates and activated prothrombin complex concentrates as prophylactic therapy in haemophilia patients with inhibitors. Haemophilia 1999;5(suppl 3):25-32
  • Ho AY, Height SE, Smith MP. Immune tolerance therapy for haemophilia. Drugs 2000;60:547-54
  • Mariani G, Kroner B. International immune tolerance registry, 1997 update. Vox Sang 1999;77(Suppl 1):25-7
  • DiMichele DM, Kroner BL. The North American Immune Tolerance Registry: practices, outcomes, outcome predictors. Thromb Haemost 2002;87:52-7
  • Lenk H. The German Registry of immune tolerance treatment in hemophilia: 1999 update. Haematologica 2000;85:45-7
  • Haya S, Lopez MF, Aznar JA, et al. Immune tolerance treatment in haemophilia patients with inhibitors: the Spanish Registry. Haemophilia 2001;7:154-9
  • Colowick AB, Bohn RL, Avorn J, et al. Immune tolerance induction in hemophilia patients with inhibitors: costly can be cheaper. Blood 2000;96:1698-702
  • Brackmann HH, Gormsen J. Massive factor-VIII infusion in haemophiliac with factor-VIII inhibitor, high responder. Lancet 1977;2:933
  • Brackmann HH, Oldenburg J, Schwaab R. Immune tolerance for the treatment of factor VIII inhibitors-twenty years' ‘Bonn protocol’. Vox Sang 1996;70(Suppl 1):30-5
  • Oldenburg J, Schwaab R, Brackmann HH. Induction of immune tolerance in haemophilia A inhibitor patients by the ‘Bonn Protocol’: predictive parameter for therapy duration and outcome. Vox Sang 1999;77(Suppl 1):49-54
  • Mauser-Bunschoten EP, Roosendaal G, van den Berg HM. Low-dose immune tolerance therapy: the Van Creveld model. Vox Sang 1996;70(Suppl 1):66-7
  • Mauser-Bunschoten EP, Nieuwenhuis HK, Roosendaal G, et al. Low-dose immune tolerance induction in hemophilia A patients with inhibitors. Blood 1995;86:983-8
  • Freiburghaus C, Berntorp E, Ekman M, et al. Tolerance induction using the Malmö treatment model 1982-1995. Haemophilia 1999;5:32-9
  • Nilsson IM, Berntorp E, Zettervall O. Induction of immune tolerance in patients with hemophilia and antibodies to factor VIII by combined treatment with intravenous IgG, cyclophosphamide, and factor VIII. N Engl J Med 1988;318:947-50
  • Berntorp E, Nilsson IM. Immune tolerance and the immune modulation protocol. Vox Sang 1996;70(Suppl 1):36-41
  • 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
  • Rocino A, Papa ML, Salerno E, et al. Immune tolerance induction in haemophilia A patients with high-responding inhibitors to factor VIII: experience at a single institution. Haemophilia 2001;7:33-8
  • Gruppo RA, Valdez LP, Stout RD. Induction of immune tolerance in patients with hemophilia A and inhibitors. Am J Pediatr Hematol Oncol 1992;14:82-7
  • Unuvar A, Kavakli K, Baytan B, et al. Low-dose immune tolerance induction for paediatric haemophilia patients with factor VIII inhibitors. Haemophilia 2008;14:315-22
  • Kroner BL. Comparison of the international immune tolerance registry and the North American immune tolerance registry. Vox Sang 1999;77(Suppl 1):33-7
  • Damiano ML, Hutter JJ Jr. Immune tolerance for haemophilia patients with inhibitors: analysis of the western United States experience. The Tri -Regional Nursing Group Haemophilia 2000;6:526-32
  • Mauser-Bunschoten EP, Rosendaal FR, Nieuwenhuis HK, et al. Clinical course of factor VIII inhibitors developed after exposure to a pasteurised Dutch concentrate compared to classic inhibitors in hemophilia A. Thromb Haemost 1994;71:703-6
  • Mariani G, Siragusa S, Kroner BL. Immune tolerance induction in hemophilia A: a review. Semin Thromb Hemost 2003;29:69-76
  • DiMichele DM, Kroner BL. Analysis of the North American Immune Tolerance Registry (NAITR) 1993-1997: current practice implications. ISTH Factor VIII/IX Subcommittee Members. Vox Sang 1999;77(Suppl 1):31-2
  • DiMichele DM, Hay CR. The international immune tolerance study: a multicenter prospective randomized trial in progress. J Thromb Haemost 2006;4:2271-3
  • Rocino A, Salerno E, Capasso F, et al. Secondary prophylaxis in severe haemophilia A: results in preventing further joint damage [abstract 1923A]. Thromb Haemost 1999;82:610
  • Unuvar A, Warrier I, Lusher JM. Immune tolerance induction in the treatment of paediatric haemophilia A patients with factor VIII inhibitors. Haemophilia 2000;6:150-7
  • Barnes C, Rivard GE, Poon MC, et al. Canadian multi-institutional survey of immune tolerance therapy (ITT): experience with the use of recombinant factor VIII for ITT. Haemophilia 2006;12:1-6
  • Rocino A, Santagostino E, Mancuso ME, et al. Immune tolerance induction with recombinant factor VIII in hemophilia A patients with high responding inhibitors. Haematologica 2006;91:558-61
  • Kreuz W, Ettingshausen CE, Auerswald G, et al. Epidemiology of inhibitors and current treatment strategies. Haematologica 2003;88:EREP04
  • Kreuz W, Escuriola-Ettingshausen C, Auerswald G. Immune tolerance induction in haemophilia A patients with inhibitors: the choice of concentrate affecting success. Haematological 2001;86(suppl 4):16-20
  • Auerswald G, Spranger T, Brackmann HH. The role of plasma-derived factor VIII/von Willebrand factor concentrates in the treatment of hemophilia A patients [abstract]. Haematological 2003;88:EREP05
  • Gringeri A, Musso R, Mazzucconi MG, et al. 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. Haemophilial 2007;13:373-9
  • Orsini F, Rothschild 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. Haematological 2005;90:1288-90
  • Behrmann M, Pasi J, Saint-Remy JM, et al. Von Willebrand factor modulates factor VIII immunogenicity: comparative study of different factor VIII concentrates in a haemophilia A mouse model. Thromb Haemost 2002;88:221-9
  • Berntorp E. Immune tolerance induction: recombinant vs. human-derived product. Haemophilial 2001;7:109-13
  • Ettingshausen CE, Kreuz W. Role of von Willebrand factor in immune tolerance induction. Blood Coagul Fibrinolysis 2005;16(Suppl 1):S27-31
  • 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
  • Suzuki T, Arai M, Amano K, et al. Factor VIII inhibitor antibodies with C2 domain specificity are less inhibitory to factor VIII complexed with von Willebrand factor. Thromb Haemost 1996;76:749-54
  • Gensana M, Altisent C, Aznar JA, et al. Influence of von Willebrand factor on the reactivity of human factor VIII inhibitors with factor VIII. Haemophilia 2001;7:369-74
  • Berntorp E. VWF/FVIII complex and the management of patient with inhibitors: from laboratory to clinical practice. Haemophilia 2007;13(Suppl 5):69-72
  • DiMichele DM. Immune tolerance: critical issues of factor dose, purity and treatment complications. Haemophilia 2006;12(Suppl 6):81-6
  • Freedman J, Rand ML, Russell O, et al. Immunoadsorption may provide a cost-effective approach to management of patients with inhibitors to FVIII. Transfusion 2003;43:1508-13
  • Knobl P, Derfler K. Extracorporeal immunoadsorption for the treatment of haemophilic patients with inhibitors to factor VIII or IX. Vox Sang 1999;77(Suppl 1):57-64
  • Rivard GE, St Louis J, Lacroix S, et al. Immunoadsorption for coagulation factor inhibitors: a retrospective critical appraisal of 10 consecutive cases from a single institution. Haemophilia 2003;9:711-6
  • Halloran PF. Immunosuppressive drugs for kidney transplantation. N Engl J Med 2004;351:2715-29
  • Cross DC, Van Der Berg HM. Cyclosporin A can achieve immune tolerance in a patient with severe haemophilia B and refractory inhibitors. Haemophilia 2007;13:111-4
  • Abdallah A, Coghlan DW, Duncan EM, et al. Rituximab-induced long-term remission in patients with refractory acquired hemophilia. J Thromb Haemost 2005;3:2589-90
  • Moschovi M, Aronis S, Trimis G, et al. Rituximab in the treatment of high responding inhibitors in severe haemophilia A. Haemophilia 2006;12:95-9
  • Escobar M, Kempton C, Ma A. Successful treatment of an autoantibody in congenital hemophilia with rituximab [abstract 3915]. Blood 2002;100:109b
  • Carcao M, St Louis J, Poon MC, et al. Rituximab for congenital haemophiliacs with inhibitors: a Canadian experience. Haemophilia 2006;12:7-18
  • Dunkley S, Lindeman R. Successful treatment of a high titre factor VIII inhibitor with rituximab alone [abstract 631]. J Thromb Haemost 2005;3(suppl 2)
  • Curry N, Stanworth S, Keeling D. The use of rituximab in two children with allo-antibodies towards factor VIII. Br J Haematol 2006;133:214-6
  • Curtin J, Misra A, Teo J, et al. Use of rituximab as an alternative strategy for the management of difficult high titre inhibitors in children with hemophilia A [abstract 30]. Haemophilia 2004;10(Suppl 3):57
  • Linde R, Escuriola-Ettinghausen C, Konigs C, et al. Rituximab as a new inhibitor elimination agent in high-titer inhibitor patients-a long-term follow-up. Haemophilia 2004;10(suppl 3):60
  • Mateo J, Badell I, Forner R, et al. Successful suppression using Rituximab of a factor VIII inhibitor in a boy with severe congenital haemophilia: an example of a significant decrease of treatment costs. Thromb Haemost 2006;95:386-7
  • Mathias M, Khair K, Hann I, et al. Rituximab in the treatment of alloimmune factor VIII and IX antibodies in two children with severe haemophilia. Br J Haematol 2004;125:366-8
  • Medeiros BC, Geraghty S, Stabler SP. Improved response to immune tolerance therapy for factor VIII inhibitor after rituximab therapy [abstract 3890]. Blood 2002;100:103b
  • Pruthi RK, Schmidt KA, Slaby JA, et al. Rituximab treatment of FVIII inhibitors in congenital hemophilia A (cHA) [abstract P652]. J Thromb Haemost 2005;3(Suppl 2)
  • Mariani G, Kroner B. Immune tolerance in hemophilia with factor VIII inhibitors: predictors of success. Haematological 2001;86:1186-93

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.