Source and purity of factor VIII products as risk factors for inhibitor development in patients with hemophilia A

Journal of Thrombosis and Haemostasis

Volumn 10, Issue 5, 2012, Pages 781-790

  Mancuso, M E ^a   Mannucci, P M ^b   Rocino, A ^c   Garagiola, I ^a   Tagliaferri, A ^d   Santagostino, E ^a  

^a Angelo Bianchi Bonomi Hemophilia and Thrombosis Center   (Italy)

^b UNIVERSITY OF MILAN   (Italy)

^c San Giovanni Bosco Hospital   (Italy)

^d UNIVERSITY HOSPITAL OF PARMA   (Italy)

Author keywords

FVIII products; Hemophilia; Inhibitors; Risk factors

Indexed keywords

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

ADULT; ARTICLE; BLOOD; BLOOD CLOTTING DISORDER; BLOOD TRANSFUSION; DRUG PURITY; FEMALE; GENE MUTATION; HAZARD RATIO; HEMOPHILIA A; HIGH RISK PATIENT; HUMAN; IMMUNOGENICITY; INCIDENCE; INHIBITOR DEVELOPMENT; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; PRIORITY JOURNAL; RETROSPECTIVE STUDY; RISK FACTOR; SENSITIVITY ANALYSIS; STATISTICAL ANALYSIS; TREATMENT RESPONSE;

ADOLESCENT; ADULT; ANTIBODIES, NEUTRALIZING; CHILD; COAGULANTS; FACTOR VIII; GENOTYPE; HEMOPHILIA A; HUMANS; ITALY; MALE; MIDDLE AGED; MUTATION; PHENOTYPE; PROPORTIONAL HAZARDS MODELS; RECOMBINANT PROTEINS; RETROSPECTIVE STUDIES; RISK ASSESSMENT; RISK FACTORS; TIME FACTORS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84860447633     PISSN: 15387933     EISSN: 15387836     Source Type: Journal    
DOI: 10.1111/j.1538-7836.2012.04691.x     Document Type: Article

References (38)

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. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003; 9: 418-35.
3. Schwaab R, Brackmann HH, Meyer C, Seehafer J, Kirchgesser M, Haack A, Olek K, Tuddenham EG, Oldenburg J. Haemophilia A: mutation type determines risk of inhibitor formation. Thromb Haemost 1995; 74: 1402-6.
4. Astermark J, Berntorp E, White GC, Kroner BL. The Malmo International Brother Study (MIBS): further support for genetic predisposition to inhibitor development in hemophilia patients. Haemophilia 2001; 7: 267-72.
5. Aledort LM, Dimichele DM. Inhibitors occur more frequently in African-American and Latino haemophiliacs. Haemophilia 1998; 4: 68.
6. Astermark J, Oldenburg J, Pavlova A, Berntorp E, Lefvert AK. 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.
7. Astermark J, Oldenburg J, Carlson J, Pavlova A, Kavakli K, Berntorp E, Lefvert AK. Polymorphisms in the TNFA gene and the risk of inhibitor development in patients with hemophilia A. Blood 2006; 108: 3739-45.
8. Astermark J, Wang X, Oldenburg J, Berntorp E, Lefvert AK; MIBS Study Group. Polymorphisms in the CTLA-4 gene and inhibitor development in patients with severe hemophilia. J Thromb Haemost 2007; 5: 263-5.
9. Pavlova A, Delev D, Lacroix-Desmazes S, Schwaab R, Mende M, Fimmers R, Astermark J, Oldenburg J. Impact of polymorphisms of the major histocompatibility complex class II, interleukin-10, tumor necrosis factor-α and cytotoxic T-lymphocyte antigen-4 genes on inhibitor development in severe hemophilia A. J Thromb Haemost 2009; 7: 2006-15.
10. Viel KR, Ameri A, Abshire TC, Iyer RV, Watts RG, Lutcher C, Channell C, Cole SA, Fernstrom KM, Nakaya S, Kasper CK, Thompson AR, Almasy L, Howard TE. Inhibitors of Factor VIII in black patients with hemophilia. N Engl J Med 2009; 360: 1618-27.
11. Lorenzo JI, Lopez A, Altisent C, Aznar JA. Incidence of factor VIII inhibitors in severe haemophilia: the importance of patient age. Br J Haematol 2001; 113: 600-3.
12. Santagostino E, Mancuso ME, Rocino A, Mancuso G, Mazzucconi MG, Tagliaferri A, Messina M, Mannucci PM. Environmental risk factors for inhibitor development in children with haemophilia A: a case-control study. Br J Haematol 2005; 130: 422-7.
13. Gouw SC, van der Bom JG, van den Berg HM. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood 2007; 109: 4648-54.
14. Escuriola Ettingshausen C, Kreuz W. Recombinant vs. plasma-derived products, especially those with intact VWF, regarding inhibitor development. Haemophilia 2006; 12(Suppl. 6): 102-6.
15. Goudemand J, Rothschild C, Demiguel V, Vinciguerrat C, Lambert T, Chambost H, Borel-Derlon A, Claeyssens S, Laurian Y, Calvez T; FVIII-LFB and Recombinant FVIII study groups. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. Blood 2006; 107: 46-51.
16. Gouw SC, van der Bom JG, Auerswald G, Escuriola Ettinghausen C, Tedgard U, van den Berg HM. Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: the CANAL cohort study. Blood 2007; 109: 4693-7.
17. Chalmers EA, Brown SA, Keeling D, Liesner R, Richards M, Stirling D, Thomas A, Vidler V, Williams MD, Young D; Paediatric Working Party of UKHCDO. Early factor VIII exposure and subsequent inhibitor development in children with severe haemophilia A. Haemophilia 2007; 13: 149-55.
18. Strauss T, Lubetsky A, Ravid B, Bashari D, Luboshitz J, Lalezari S, Misgav M, Martinowitz U, Kenet G. Recombinant factor concentrates may increase inhibitor development: a single centre cohort study. Haemophilia 2011; 17: 625-9.
19. MacLean PS, Richards M, Williams M, Collins P, Liesner R, Keeling DM, Yee T, Will AM, Young D, Chalmers EA; Paediatric Working Party of UKHCDO. Treatment related factors and inhibitor development in children with severe haemophilia A. Haemophilia 2011; 17: 282-7.
20. Iorio A, Halimeh S, Holzhauer S, Goldenberg N, Marchesini E, Marcucci M, Young G, Bidlingmaier C, Brandao LR, Ettingshausen CE, Gringeri A, Kenet G, Knöfler R, Kreuz W, Kurnik K, Manner D, Santagostino E, Mannucci PM, Nowak-Göttl U. Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review. J Thromb Haemost 2010; 8: 1256-65.
21. Kasper CK, Aledort L, Aronson D, Counts R, Edson JR, van Eys J, Fratantoni J, Green D, Hampton J, Hilgartner M, Levine P, Lazerson J, McMillan C, Penner J, Shapiro S, Shulman NR. A more uniform measurement of factor VIII inhibitors. Thromb Diath Haemorrh 1975; 34: 612.
22. Verbruggen B, Novakova I, Wessels H, Boezeman J, van den Berg HM, Mauser-Bunschoten E. The Nijmegen modification of the Bethesda assay for factor VIII:C inhibitors: improved specificity and reliability. Thromb Haemost 1995; 73: 247-51.
23. Rothschild C, Gill J, Scharrer I, Bray G. Transient inhibitors in the Recombinate PUP study. Thromb Haemost 2000; 84: 145-6.
24. Oldenburg J, Pavlova A. Genetic risk factors for inhibitors to factor VIII and IX. Haemophilia 2006; 12(Suppl. 6): 15-22.
25. Addiego J, Kasper C, Abilgaard C, Hilgartner M, Lusher J, Glader B, Aledort L. Frequency of inhibitor development in haemophiliacs treated with low-purity factor VIII. Lancet 1993; 342: 462-4.
26. Morfini M. In vitro characteristics of highly purified factor VIII concentrates. Ann Hematol 1991; 63: 123-5.
27. von Elm E, Altman DG, Egger M, Pocock SJ, Gøtzsche PC, Vandenbroucke JP; STROBE Initiative. The strengthening the reporting of observational studies in epidemiology (STROBE) statement: guidelines for reporting observational studies. Lancet 2007; 370: 1453-7.
28. Mannucci PM. Back to the future: a recent history of haemophilia treatment. Haemophilia 2008; 14(Suppl. 3): 10-8.
29. Santagostino E, Mannucci PM. Guidelines on replacement therapy for haemophilia and inherited coagulation disorders in Italy. Haemophilia 2000; 6: 1-10.
30. Calvez T, Laurian Y, Goudemand J. Associations between type of product and inhibitors in previously untreated patients (PUPs) with severe hemophilia: switches and particular products can disturb analysis. Blood 2007; 110: 1073-4.
31. van der Bom JG, Gouw SC, van den Berg HM. Plasma-derived or recombinant factor VIII products and inhibitors in previously untreated patients with severe hemophilia. Blood 2007; 110: 1074-5.
32. Dasgupta S, Repessé Y, Bayry J, Navarrete AM, Wootla B, Delignat S, Irinopoulou T, Kamaté C, Saint-Remy JM, Jacquemin M, Lenting PJ, Borel-Derlon A, Kaveri SV, Lacroix-Desmazes S. VWF protects FVIII from endocytosis by dendritic cells and subsequent presentation to immune effectors. Blood 2007; 109: 610-2.
33. Qadura M, Waters B, Burnett E, Chegeni R, Bradshaw S, Hough C, Othman M, Lillicrap D. Recombinant and plasma-derived factor VIII products induce distinct splenic cytokine microenvironments in hemophilia A mice. Blood 2009; 114: 871-80.
34. 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.
35. 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.
36. Ghio M, Contini P, Ottonello L, Arduino N, Gringeri A, Indiveri F, Dallegri F, Puppo F. Effect of clotting factors concentrates on lymphocyte and neutrophil function in vitro. Thromb Haemost 2003; 89: 365-73.
37. Kurnik K, Bidlingmaier C, Engl W, Chehadeh H, Reipert B, Auerswald G. New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor development. Haemophilia 2010; 16: 256-62.
38. Auerswald G, Bidlingmaier C, Kurnik K. Early prophylaxis/FVIII tolerization regimen that avoids immunological danger signals is still effective in minimizing FVIII inhibitor developments in previously untreated patients - long-term follow-up and continuing experience. Haemophilia 2012; 18: e1-41.