Inhibitors to Factor VIII/IX: Immune Tolerance

Textbook of Hemophilia: Second Edition

Volumn , Issue , 2010, Pages 66-74

  Dimichele, Donna ^a  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

Author keywords

Central venous access complications of immune tolerance induction; Host factors and their role in immune tolerance induction outcome; Immune modulation; Immune tolerance factor VIII inhibitors; Immune tolerance induction outcome; Inhibitor associated morbidity, limited options, antibody eradication goal of inhibitor management; Inhibitors to factor VIII IX immune tolerance; North American Immune Tolerance Registry (NAITR); Strategy for antigen specific tolerance to factor VIII (FVIII) immune tolerance induction (ITI) therapy; Treatment associated factors with effect on ITI outcome

Indexed keywords

EID: 84885823567     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9781444318555.ch10     Document Type: Chapter

References (49)

1. Brackmann HH, Gormsen J. Massive factor-VIII infusion in haemophiliac with factor-VIII inhibitor, high responder. Lancet 1977; 2: 993.
2. DiMichele D. Immune tolerance therapy for factor VIII inhibitors: Moving from empiricism to an evidence-based approach. J Thromb Haemost 2007 5 ( Suppl. 1 ): 143-150.
3. Mariani G, Scheibel E, Nogao T, et al. Immune tolerance as treatment of alloantibodies to factor VIII in hemophilia. The International Registry of Immunetolerance Protocols. Semin Hematol 1994; 31 ( Suppl. 4 ): 62-64.
4. DiMichele D, Kroner B. Factor VIII/IX Subcommittee of the International Society for Thrombosis and Hemostasis. The maintenance of tolerance after successful immune tolerance induction in haemophilia A and B: The North American Registry. Factor VIII/IX Subcommittee of the International Society for Thrombosis and Haemostasis. Haematologica 2000; 85 ( Suppl. 10 ): 40-44.
5. DiMichele DM, Hay CRM. The international immune tolerance study: A multicenter prospective randomised trial in progress. J Thromb Haemost 2006; 4: 2271-3000.
6. Astermark J, Morado M, Rocino A, et al. Current European practice in immune tolerance induction therapy in patients with haemophilia and inhibitors. Haemophilia 2006; 12: 363-71.
7. DiMichele DM, Hoots WK, Pipe SW, Rivard GE, Santagostino E. International workshop on immune tolerance induction: Consensus recommendations. Haemophilia 2007; 13: ( Suppl. 1 ): 1-22.
8. vanHelden PMW, van den Berg HM, Gouw SC, et al. IgG subclasses of anti-FVIII antibodies during immune tolerance induction in patients with hemophilia A. Br J Haematol 2008; 142: 644-52.
9. Lenk H, ITT Study Group. The German Registry of immune tolerance treatment in hemophilia - 1999 update. Haematologica 2000; 10 ( Suppl. ): 45-7.
10. DiMichele DM, Kroner B, and the North American Immune Tolerance Study Group. The North American Immune Tolerance Registry: Practices, outcomes, outcome predictors. Thromb Haemost 2002; 87: 52-57.
11. Rocino A, Santagostino E, Mancuso ME, Mannucci PM. Immune tolerance induction with recombinant factor VIII in haemophilia A patients with high responding inhibitors. Haematologica 2006; 91: 558-61.
12. Oldenburg J, Schwaab R, Brackmann HH. Induction of immune tolerance in haemophilia A. Inhibitor patients by the " Bonn Protocol ": Predictive Parameters for Therapy, Duration & Outcome. Vox Sang 1999; 77 ( Suppl. 1 ): 49-54.
13. Mariani G, Kroner B. Immune tolerance in haemophilia with factor VIII inhibitors: Predictors of success. Haematologica 2001; 86: 1186-93.
14. Kroner BL. Comparison of the International Immune Tolerance Registry and the North American Immune Tolerance Registry. Vox Sang 1999; 77 ( Suppl. 1 ): 33-7.
15. Ettingshausen CE, Kreuz W. Role of von Willebrand factor in immune tolerance induction. Blood Coagul Fibrinolysis 2005; 16: ( Suppl. 1 ): S27-31.
16. 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.
17. Greninger DA, Saint-Remy JM, Jacquemin M, Benhida A, DiMichele DM. 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 profi le. Haemophilia 2008; 2: 295-302.
18. 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.
19. 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; 1: 50-55.
20. Goudemand J. Inhibitor development in haemophilia A: The role of von Willebrand factor/factor VIII concentrates. Haemophilia 2007; 13 ( Suppl. 5 ): 47-51.
21. Nilsson IM, Berntorp E, Zettervall O. Induction of immune tolerance in patients with haemophilia and antibodies to factor VIII by combined treatment with intravenous IgG, cyclophosphamide, and factor VIII. N Engl J Med 1988; 318: 947-50.
22. Freiburghaus C, Berntorp E, Ekman M, Gunnarsson M, Kjellberg BM, Nilsson IM. Tolerance induction using the Malm ö treatment model 1982-1995. Haemophilia 1999; 5: 32-9.
23. PW Collins. Novel therapies for immune tolerance in haemophilia A. Haemophilia 2006; 12 ( Suppl. 6 ): 94-101.
24. Ewenstein BM, Valentino LA, Journeycake JM, et al. Consensus recommendations for use of central venous access devices in haemophilia. Haemophilia 2004; 10: 629-48.
25. Mancuso ME, Mannucci PM, Sartori A, Agliardi A, Santagostino E. Feasibility of prophylaxis and immune tolerance induction regimens in haemophilic children using fully implantable central venous catheters. Br J Haematol 2008; 141: 689-95.
26. Santagostino E, Gringeri A, Berardinelli L, Beretta C, Muca-Perja M, Mannucci PM. Long-term safety and feasibility of arteriovenous fi stulae as vascular accesses in children with haemophilia: A prospective study. Br J Haematol 2003; 123: 502-6.
27. Soucie JM, Cianfrini C, Janco RL, et al. Joint-range-of motion limitations among young males with haemophilia: Prevalence and risk factors. Blood 2004; 103: 2467-73.
28. Manco-Johnson ML, Funk S, Jacobson L, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
29. Kreuz W, Escuriola-Ettingshausen C, Mentzer D, et al. Factor VIII inhibitor bypass activity (FEIBA) for prophylaxis during immune tolerance induction (ITI) in patients with high-responding inhibitors [Abstract]. Blood 2000; 96: 266a. Abstract 1141.
30. Carcao MD, Connolly BL, Chait P, et al. Central venous catheter-related thrombosis presenting as superior vena cava syndrome in a haemophilic patient with inhibitors. Haemophilia 2003; 5: 578-83.
31. Konkle BA, Ebbesen LS, Erhardtsen E, et al. Randomised, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2007; 9: 1904-13.
32. Hay CRM, Brown S, Collins PW, Keeling DM, Liesner R. The diagnosis and management of factor VIII and IX inhibitors: A guideline from the United Kingdom Centre Doctors Organisation. Br J Haematol 2006; 133: 591-605.
33. 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.
34. Warrier I. Inhibitors in haemophilia B. In: Lee C, Berntorp E, Hoots KW (eds.) Textbook of Hemophilia. Oxford: Blackwell Publishers, 2005; 97-100
35. Ewenstein BM, Takemoto C, Warrier I, et al. Nephrotic syndrome as a complication of immune tolerance in hemophilia B. Blood 1997; 89: 1115-16.
36. DiMichele D. ( 2008 ) The North American immune tolerance registry: Contributions to the thirty-year experience with immune tolerance therapy. Haemophilia 2009; 15: 320-8.
37. Reipert BM, van den Helden PMW, Schwarz HP, Causl C. Mechanisms of action of immune tolerance induction against factor VIII in patients with congenital haemophilia A and factor VIII inhibitors. Br J Haematol 2006; 136: 12-25.
38. Pipe SW. The promise and challenges of bioengineered recombinant clotting factors. J Thromb Haemost 2005; volume: 1692-701.
39. Dobrzynski E, Fitzgerald JC, Cao O, Mingozzi F, Wang L, Hertzog RW. Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induces regulatory T cells. Proc Natl Acad Sci USA 2006; 103: 4592-7.
40. Ashrani AA, Reding MT, Shet A, et al. Successful liver transplantation in a patient with severe haemophilia A and a high titre factor VIII inhibitor. Haemophilia 2004; 10: 735-7.
41. 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 ): 30-35.
42. Orsini F, Rothschild C, Beurrier P, Faradji A, Goudemand J, Polack B. Immune tolerance induction with highly purifi ed plasma-derived factor VIII containing von Willebrand factor in hemophilia A patients with high-responding inhibitors. Haematologica 2005; 90: 1288-90.
43. Smith MP, Spence KJ, Waters EL, et al. Immune tolerance therapy for hemophilia A patients with acquired factor VIII alloantibodies: comprehensive analysis of experience at a single institution. Thromb Haemost 1999; 81: 35-88.
44. Luster JM. Summary of clinical experience with recombinant factor VIII products - Kogenate. Ann Hematol 1994; 68 ( Suppl. 3 ): S3-6.
45. Bray GL, Gomperts ED, Courter S, et al. A multicenter study of recombinant factor VIII (Recombinate): safety, effi cacy, and inhibitor risk in previously untreated patients with hemophilia A. The Recombinate Study Group. Blood 1994; 83: 2428-35.
46. 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.
47. Courter SG, Bedrosian CL. Clinical evaluation of B-domain deleted recombinant factor VIII in previously untreated patients. Semin Hematol 2001; 38 ( Suppl. 4 ): 52-9.
48. Barnes C, Rivard GE, Poon MC, et al. Inhibitor Subcommittee of Association of Heuophilia Clinic Directors of Canada. Canadian multi-institutional survey of immune tolerance therapy (ITT) - experience with the use of recombinant factor VIII for ITT. Haemophilia 2006; 12: 1-6.
49. Rocino A, Papa ML, Salerno E, Capasso F, Miraglia E, deBiasi R. Immune tolerance induction in hemophilia A patients with high-responding inhibitors to factor VIII: experience at a single institution. Haemophilia 2001; 7: 33-8.