Recombinant vs. plasma-derived products, especially those with intact VWF, regarding inhibitor development

Haemophilia

Volumn 12, Issue SUPPL. 6, 2006, Pages 102-106

  Ettingshausen, Carmen Escuriola ^a   Kreuz, W ^a  

^a UNIVERSITY HOSPITAL FRANKFURT   (Germany)

Author keywords

FVIII concentrates; Haemophilia A; Inhibitor; Von Willebrand factor

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING INHIBITOR; RECOMBINANT BLOOD CLOTTING FACTOR 8; VON WILLEBRAND FACTOR;

ARTICLE; DATA ANALYSIS; DISEASE SEVERITY; HEMOPHILIA A; HUMAN; HYPOTHESIS; IMMUNOGENICITY; INCIDENCE; OBSERVATIONAL STUDY; PRIORITY JOURNAL; PROSPECTIVE STUDY; RISK FACTOR; STATISTICAL ANALYSIS; SYSTEMATIC REVIEW;

BLOOD COAGULATION FACTOR INHIBITORS; CHILD; CHILD, PRESCHOOL; COAGULANTS; FACTOR VIII; HEMOPHILIA A; HUMANS; RECOMBINANT PROTEINS; RISK FACTORS; VON WILLEBRAND FACTOR;

EID: 33751010515     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2006.01373.x     Document Type: Article

References (30)

1. Kreuz W, Auerswald G, Budde U, Lenk H and the GTH-PUP-Study Group. In: Scharrer I, Schramm W eds. Inhibitor development in previously treated patients (PUPs) with haemophilia A and B. A prospective multicentre study of the German, Swiss and Austrian Society of Thrombosis and Haemostasis Research (GTH). 35th Haemophilia Symposium, Hamburg 2004. Heidelberg: Springer Verlag, 2005: 34-7.
2. Schwaab R, Brackmann HH, Meyer C et.al. Haemophilia A: Mutation type determines risk of inhibitor formation. Thromb Haemost 1995; 74: 1402-6.
3. Aledort LM, Michele DM. Inhibitors occur more frequently in African-American and Latino haemophiliacs. Haemophilia 1998; 4: 68.
4. Astermark J, Oldenburg J, Pavlova A, Berntorp E, Lefvert AK, MIBS Study Group. Polymorphisms in the IL-10 but not in the IL-1ß and IL-4 genes are associated with inhibitor development in patients with haemophilia A. Blood 2006; 107: 3167-72.
5. Lorenzo JI, Lopez A, Altisent C, Aznar JA. Incidence of factor VIII inhibitors in severe haemophilia: The importance of age. Br J Haematol 2001; 113: 600-3.
6. Van der Bom JG, Mauser-Bunschoten EP, Fischer K, van den Berg HM. Age at first treatment and immune tolerance to factor VIII in severe hemophilia. Thromb Haemost 2003; 89: 475-9.
7. 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.
8. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: A systematic review. Haemophilia 2003; 9: 418-35.
9. Guerois C, Laurian Y, Rothschild C et.al. Incidence of factor VIII inhibitor development in severe hemophilia A patients treated only with one brand of highly-purified plasma-derived concentrate. Thromb Haemost 1995; 73: 215-8.
10. Peerlinck K, Rosendaal FR, Vermylen J. Incidence of inhibitor development in a group of young hemophilia A patients treated exclusively with lyophylized cryoprecipitate. Blood 1993; 81: 3332-5.
11. Yee TT, Williams MD, Hill FG, Lee CA, Pasi KJ. Absence of inhibitors in previously untreated patients with severe haemophilia A after exposure to a single intermediate purity factor VIII product. Thromb Haemost 1997; 78: 1027-9.
12. Schimpf K, Schwarz P, Kunschak M. Zero incidence of inhibitors in previously untreated patients who received intermediate purity factor VIII concentrate or factor IX complex. Thromb Haemost 1995; 73: 553-5.
13. Bray GL, Gomperts ED, Courter SG. A multicenter study of recombinant factor VIII (recombinate): Safety, efficacy, and inhibitor risk in previously untreated patients. Blood 1997; 83: 2428-35.
14. Lusher JM, Arkin S, Abildgaard CF, Schwartz RS. Recombinant factor VIII for the treatment of previously untreated patients with haemophilia A. Safety, efficacy and development of inhibitors. Kogenate previously untreated patient study group. N Engl J Med 1993; 328: 453-9.
15. 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.
16. Courter SG, Bedrosian CL. Clinical evaluation of B-domain deleted recombinant factor VIII in previously untreated patients. Semin Hematol 2001; 38: 52-9.
17. Kreuz W, Gill JC, Rothschild C et.al. Full-length sucrose-formulated recombinant factor VIII for treatment of previously untreated or minimally treated young children with severe haemophilia A: Results of an international clinical investigation. Thromb Haemost 2005; 3: 457-67.
18. Gringeri A, Monzini M, Tagariello G, Scaraggi FA, Mannucci PM, the EMOCLOT15 Study Members. Occurrence of inhibitors in previously untreated or minimally treated patients with haemophilia A after exposure to a plasma-derived solvent-detergent factor VIII concentrate. Haemophilia 2006; 12: 128-32.
19. Goudemand J, Rothschild C, Demiguel V et.al. Influence of the type of factor VIII concentrate on the incidence of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe haemophilia A. Blood 2006; 107: 46-51.
20. Rokicka-Milewska R, Klukowska A, Dreger B, Beer HJ. Incidence of factor VIII inhibitor development in previously untreated haemophilia A patients after exposure to a double virus inactivated factor VIII concentrate. Ann Haematol 1999; 78(Suppl. 1): 1-7.
21. Saenko EL, Ananyeva NM, Kouiavskaia DV et.al. Haemophilia A: Effects of inhibitory antibodies on factor VIII functional interactions and approaches to prevent their action. Haemophilia 2002; 8: 1-11.
22. 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.
23. Suzuki T, Arai M, Amano K, Kagawa K, Fukutake K. Factor VIII inhibitor antibodies with C2 domain specifity are less inhibitory to factor VIII complexed with von Willebrand factor. Thromb Haemost 1996; 76: 749-54.
24. Jacquemin MG, Saint-Remy JM. Factor VIII immunogenicity. Haemophilia 1998; 4: 552-7.
25. 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.
26. Ananyeva NM, Lacroix-Desmazes S, Hauser CA et.al. Inhibitors in hemophilia A: Mechanisms of inhibition, managemant and perspectives. Blood Coagul Fibrinolysis 2004; 15: 109-24.
27. Reipert BM, Schoppmann A, Schwarz HP. A caution on the use of murine hemophilia models for comparative immunogenicity studies of FVIII products with different protein compositions. Thromb Haemost 2003; 89: 1110-2.
28. Hodge G, Flower R, Han P. Effect of factor VIII concentrate on leucocyte cytokine production: Characterisation of TGF-beta as an immunomodulatory component in plasma derived factor VIII concentrate. Br J Haematol 1999; 106: 784-91.
29. Hodge G, Saxon B, Revesz T. Effect of factor VIII concentrate on leucocyte cytokine receptor expression in vitro: Relevance to inhibitor formation and tolerance induction. Haemophilia 2006; 12: 133-9.
30. Hodge G, Han P. FVIII concentrate inhibits T-helper-type 2 (Th2) cytokine production in vitro: Relevance to inhibitor antibody formation. Haemophilia 2001; 7: 490-6.