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

Volumn 13, Issue 4, 2007, Pages 373-379

  Gringeri, Alessandro ^a   Musso, R ^b   Mazzucconi, M G ^c   Piseddu, G ^d   Schiavoni, M ^e   Pignoloni, P ^c   Mannucci, P M ^a   Bernasconi, S ^f   Brito, D ^f   Castaman, G ^f   Coppola, A ^f   Di Minno, G ^f   Gamba, G ^f   Iannaccaro, P G ^f   Landolfi, R ^f   Perja, M Muça ^f   Prieto, M ^f   Santagostino, E ^f   Schinco, P C ^f   Villar, A ^f  

^a UNIVERSITY OF MILAN   (Italy)

^b Ferrarotto Hospital   (Italy)

^c SAPIENZA UNIVERSITY OF ROME   (Italy)

^d SS Annunziata Hospital   (Italy)

^e UNIVERSITY OF BARI MEDICAL SCHOOL   (Italy)

^f NONE

Author keywords

Haemophilia A; Immune tolerance; Inhibitors; Rescue treatment; VWF

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; BERIATE P; BLOOD CLOTTING FACTOR; BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 8 INHIBITOR; BLOOD CLOTTING FACTOR 8 VON WILLEBRAND FACTOR COMPLEX CONCENTRATE; EMOCLOT; EPITOPE; FANHDI; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 8; UNCLASSIFIED DRUG; VON WILLEBRAND FACTOR;

ADOLESCENT; ADULT; ARTICLE; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; DISEASE SEVERITY; DRUG EFFICACY; DRUG HALF LIFE; DRUG MEGADOSE; DRUG METABOLISM; DRUG PURITY; DRUG RESPONSE; DRUG TREATMENT FAILURE; DRUG WITHDRAWAL; FOLLOW UP; HEMOPHILIA A; HIGH RISK PATIENT; HUMAN; IMMUNOLOGICAL TOLERANCE; MULTICENTER STUDY; OUTCOME ASSESSMENT; PRIORITY JOURNAL; PROGNOSIS; PROTEIN BLOOD LEVEL; TREATMENT DURATION;

ADOLESCENT; ADULT; BLOOD COAGULATION FACTOR INHIBITORS; CHILD; CHILD, PRESCHOOL; DOSE-RESPONSE RELATIONSHIP, DRUG; HEMOPHILIA A; HUMANS; IMMUNE TOLERANCE; IMMUNOSUPPRESSION; MIDDLE AGED; RISK ASSESSMENT; VON WILLEBRAND FACTOR;

EID: 34447287337     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2007.01484.x     Document Type: Article

References (37)

1. 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.
2. Wight J, Paisley S, Knight C. Immune tolerance induction in patients with haemophilia A with inhibitors: A systematic review. Haemophilia 2003; 9: 436-63.
3. Brackmann HH, Gormsen J. Massive factor-VIII infusion in haemophiliac with factor-VIII inhibitor, high responder. Lancet 1977; 2: 933.
4. Di Michele DM. Immune tolerance: A synopsis of the international experience. Haemophilia 1998; 4: 568-73.
5. Lenk H. The German Registry of immune tolerance treatment in hemophilia - 1999 update. Haematologica 2000; 85 (Suppl. 10): 45-7.
6. Mariani G, Kroner B. Immune tolerance in hemophilia with factor VIII inhibitors: Predictors of success. Haematologica 2001; 86: 1186-93.
7. 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.
8. Kreuz W. Immune tolerance therapy in paediatric haemophiliacs with factor VIII inhibitors: 14 years follow-up. Haemophilia 1995; 1: 24-32.
9. DiMichele D. Immune tolerance therapy dose as an outcome predictor. Haemophilia 2003; 9: 382-6.
10. Hay CR. Hemophilia treatment. Immune tolerance induction: Prospective clinical trials. Haematologica 2000; 85 (Suppl. 10): 52-5.
11. Federici AB. The factor VIII/Von Willebrand Factor complex: From physio-pathology to clinical practice. Haematologica 2001; 86 (Suppl. 4): 6-15.
12. 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.
13. Mannucci PM. Von Willebrand factor: A prima ballerina on two different stages. Semin Hematol 2005; 42: 1-4.
14. Amano K, Arai M, Koshihara K et al. Autoantibody to factor VIII that has less reactivity to factor VIII/von Willebrand factor complex. Am J Hematol 1995; 49: 310-7.
15. 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.
16. Berntorp E, Ekman M, Gunnarsson M, Nilsson IM. Variation in factor VIII inhibitor reactivity with different commercial factor VIII preparations. Haemophilia 1995; 2: 95-9.
17. 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.
18. Kallas A, Talpsep T. Willebrand factor in factor VIII concentrates protects against neutralization by factor VIII antibodies of haemophilia A patients. Haemophilia 2001; 7: 375-80.
19. Suzuki T, Arai M, Amano K, Kagawa K, Fukutake K. 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.
20. 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: 221-9.
21. Orsini F, Rotschild C, Beurrier P, Faradji A, Goudemand J, Polack B. 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.
22. Auerswald G, Spranger T, Brackmann HH. The role of plasma-derived factor VIII/von Willebrand factor concentrates in the treatment of hemophilia A patients. Haematologica 2003; 88: EREP05.
23. Kreuz W, Ettingshausen CE, Auerswald G et al. Epidemiology of inhibitors and current treatment strategies. Haematologica 2003; 88: EREP04.
24. Ghio M, Contini P, Ottonello L et al. Effect of clotting factors concentrates on lymphocyte and neutrophil function in vitro. Thromb Haemost 2003; 89: 365-73.
25. Thorpe R, Dilger P, Dawson NJ, Barrowcliffe TW. Inhibition of interleukin-2 secretion by factor VIII concentrates: A possible cause of immunosuppression in haemophiliacs. Br J Haematol 1989; 71: 387-91.
26. Verbruggen B, Novakova I, Wessels H, Boezeman J, Van den Berg M, Mauser-Bunschoten E. The Nijmegen modification of the Bethesda assay for Factor IIII:C inhibitors: improved specificity and reliability. Thomb Haemostas 1995; 73: 247-51.
27. Fulcher CA, de Lechner KG, Mhoney S. Immunoblot analysis shows changes in FVIII inhibitors chain specificity in factor VIII inhibitors patients over time. Blood 1988; 72: 1348-56.
28. 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.
29. Sukhu K, Keeling DM, Giangrande PL. Variation in inhibitor reactivity in acquired haemophilia A with different concentrates. Clin Lab Haematol 2000; 22: 287-90.
30. Eibl MM, Ahmad R, Wolf HM, Linnau Y, Götz E, Mannhalter JW. A component of factor VIII preparations which can be separated from factor VIII activity down modulates human monocyte functions. Blood 1987; 69: 1153-60.
31. Vermont-Desrochers C, Rigal D, Blourde C, Bernaud J. Immunosuppressive property of a very high purity antihaemophilic preparation: A low molecular weight component inhibits an early step of PHA induced cell activation. Br J Haematol 1992; 80: 370-7.
32. Hodge G, Han P. Effect of factor VIII concentrate on antigen-presenting cell (APC)/T-cell interactions in vitro: Relevance to inhibitor formation transforming growth factor-beta as a contaminant in factor VIII concentrates: A possible link with immunosuppressive effects in hemophiliacs. Blood 1994; 84: 2021-30.
33. Hodge G, Lloyd JV, Hodge S, Story C, Han P. Functional lymphocyte immunophenotypes observed in thalassaemia and haemophilia patients receiving current blood product preparations. Br J Haematol 1999; 105: 817-25.
34. Hodge G, Flower R, Han P. Effect of factor VIII concentrate on leukocyte cytokine production: Characterization of TGF-beta as an immunomodulatory component on plasmaderived factor VIII concentrate. Br J Haematol 1999; 106: 784-91.
35. Pearson HJL, Stirling D, Ludlam CA, Steel CM. TGF- β is not the principal immunosuppressive component in coagulation factor concentrates. Br J Haematol 1999; 106: 971-9.
36. Wadhwa M, Barrowcliffe T, Thorpe R. Immunological effects of factor VIII concentrates: Characterization of transforming growth factor β as an immunomodulatory contaminant in factor VIII concentrates. Br J Haematol 2000; 109: 895-905.
37. Hodge G, Han P. Effect of intermediate-purity factor VIII (FVIII) concentrate on lymphocyte proliferation and apoptosis: Transforming growth factor- β is a significant immunomodulatory component of FVIII. Br J Haematol 2001; 115: 376-81.