Alloantibodies in previously untreated hemophilia A patients: the role of environmental factors

References

• Coppola C, Santoro C, Tagliaferri A, Franchini M, Di Minno G. Understanding inhibitor development. Haemophilia. 2010;16 (Suppl 1):13–9.
   PubMed Web of Science ®Google Scholar
• Franchini M, Coppola A, Molinari AC, Santoro C, Schinco P, Speciale V, et al. Forum on: the role of recombinant factor VIII in children with severe haemophilia A. Haemophilia. 2009;15:578–86.
   PubMed Web of Science ®Google Scholar
• Gomperts ED. The need for previously untreated patient population studies in understanding the development of factor VIII inhibitors. Haemophilia. 2006;12:573–8.
   PubMed Web of Science ®Google Scholar
• Lee CA, Lillicrap D, Astermark J. Inhibitor development in hemophiliacs: the roles of genetic versus environmental factors. Semin Thromb Haemost. 2006;32 (Suppl. 2):10–14.
   PubMed Web of Science ®Google Scholar
• Cox-Gill J. The role of genetics in inhibitor formation. Thromb Haemost. 1999;82:500–4.
   PubMed Web of Science ®Google Scholar
• Astermark J. Overview of inhibitors. Semin Hematol. 2006;43 (2 Suppl.):S3–S7.
   PubMed Web of Science ®Google Scholar
• Gouw SC, van den Berg HM. The multifactorial etiology of inhibitor development in hemophilia: genetics and environment. Semin Thromb Hemost. 2009;35:723–34.
   PubMed Web of Science ®Google Scholar
• Matzinger P. The danger model: a renewed sense of self. Science. 2002;8:76–82.
   Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• van der Bom JG, Mauser-Bunschoten P, 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.
   PubMed Web of Science ®Google Scholar
• Santagostino E, Mancuso ME, Rocino A, Mancuso G, Mazzucconi MG, Tagliaferri 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.
   PubMed Web of Science ®Google Scholar
• Goudemand J, Rothschild C, Demiguel V, Vinciguerrat C, Lambert T, Chambost H, et al. 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.
   PubMed Web of Science ®Google Scholar
• Chalmers EA, Brown SA, Keeling D, Liesner R, Richards M, Stirling D, et al. Early factor VIII exposure and subsequent inhibitor development in children with severe haemophilia A. Haemophilia. 2007;13:149–55.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Gouw SC, van den Berg HM, le Cessie S, van der Bom JG. Treatment characteristics and the risk of inhibitor development: a multicenter cohort study among previously untreated patients with severe hemophilia A. J Thromb Haemost. 2007;5:1383–90.
   PubMed Web of Science ®Google Scholar
• Maclean PS, Richards M, Williams M, Collins P, Liesner R, Keeling DM, et al. Paediatric Working Party of UKHCDO. Treatment related factors and inhibitor development in children with severe haemophilia A. Haemophilia. 2011;17:282–7.
   PubMed Web of Science ®Google Scholar
• Ter Avest PC, Fisher K, Mancuso ME, Santagostino E, Yuste VJ, van den Berg HM, et al. CANAL Study Group. Risk stratification for inhibitor development at first treatment for severe hemophilia A: a tool for clinical practice. J Thromb Haemost. 2008;6:2048–54.
   PubMed Web of Science ®Google Scholar
• Eckhardt CL, van der Bom JG, van der Naald M, Peters M, Kamphuisen PW, Fijnvandraat K. Surgery and inhibitor development in hemophilia A: a systematic review. J Thromb Haemost. 2011;9:1948–58.
   PubMed Web of Science ®Google Scholar
• Yee TT, Beeton K, Griffioen A, Harrington C, Miners A, Lee CA, et al. Experience of prophylaxis treatment in children with severe haemophilia. Haemophilia. 2002;8:76–82.
   PubMed Web of Science ®Google Scholar
• Morado M, Villar A, Jiménez Yuste V, Quintana M, Hernandez Navarro F. Prophylactic treatment effects on inhibitor risk: experience in one centre. Haemophilia. 2005;11:79–83.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Gouw SC, van den Berg M, van der Bom J. Factor VIII prophylaxis and inhibitor development in previously untreated patients with severe hemophilia A: the RODIN study. Haemophilia. 2012;18(Suppl. 3):89, (abstract).
   PubMedGoogle Scholar
• Franchini M, Lippi G. VWF-containing FVIII concentrates and inhibitors in hemophilia a: a critical literature review. Thromb Haemost. 2010;104:931–40.
   PubMed Web of Science ®Google Scholar
• Verbruggen B, van Heerde W, Budde U. Methodological shortcomings in assessment of factor VIII concentrate inhibition. Haemophilia. 2007;13:680–1.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Dasgupta S, Repesse Y, Bayry J, Navarrete AM, Wootla B, Delignat S, et al. VWF protects FVIII from endocytosis by dendritic cells and subsequent presentation to immune effectors. Blood. 2007;109:610–2.
   PubMed Web of Science ®Google Scholar
• Pfistershammer K, Stockl J, Siekmann J, Turecek PL, Schwarz HP, Reipert BM. Recombinant factor VIII and factor VIII–von Willebrand factor complex do not present danger signals for human dendritic cells. Thromb Haemost. 2006;96:309–16.
   PubMed Web of Science ®Google Scholar
• Mauser-Bunschoten EP, van der Bom JG, Bongers M, Twijnstra M, Roosendaal G, Fischer K, et al. Purity of factor VIII product and incidence of inhibitors in previously untreated patients with haemophilia A. Haemophilia. 2001;7:364–8.
   PubMed Web of Science ®Google Scholar
• Kreuz W, Ettingshausen CE, Zyschka A, Oldenburg J, Saguer IM, Ehrenforth S, et al. Inhibitor development in previously untreated patients with hemophilia A: a prospective long-term follow-up comparing plasma-derived and recombinant products. Semin Thromb Hemost. 2002;28:285–90.
   PubMed Web of Science ®Google Scholar
• Kreuz W, Ettingshausen CE, Auerswald G, Saguer IM, Becker S, Funk M, et al. Epidemiology of inhibitors and current treatment strategies. Haematologica. 2003;88:pEREP04.
   PubMedGoogle Scholar
• Gouw SC, van der Bom JG, Auerswald G, Ettinghausen CE, Tedgård 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.
   PubMed Web of Science ®Google Scholar
• Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia. 2003;9:418–35.
   PubMed Web of Science ®Google Scholar
• Scharrer I, Ehrlich HJ. Reported inhibitor incidence in FVIII PUP studies: comparing apples with oranges? Haemophilia. 2004;10:197–8.
   PubMed Web of Science ®Google Scholar
• Iorio A, Halimeh S, Holzhauer S, Goldenberg N, Marchesini E, Marcucci M, et al. 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.
   PubMed Web of Science ®Google Scholar
• Franchini M, Tagliaferri A, Mengoli C, Cruciani M. Cumulative inhibitor incidence in previously untreated patients with severe hemophilia A treated with plasma-derived versus recombinant factor VIII concentrates: a critical systematic review. Crit Rev Oncol Hematol. 2012;81:82–93.
   PubMed Web of Science ®Google Scholar
• Lusher JM, Salzman PM. Viral safety and inhibitor development associated with factor VIIIC ultra-purified from plasma in haemophiliacs previously unexposed to factor VIII C concentrates. Semin Hematol. 1990;27 (Suppl. 2):1–7.
   PubMed Web of Science ®Google Scholar
• Lusher JM. Viral safety and inhibitor development associated with monoclonal antibody-purified FVIII:C. Ann Hematol. 1991;63:138–41.
   PubMed Web of Science ®Google Scholar
• Ehrenforth S, Kreuz W, Scharrer I, Linde R, Funk M, Güngör T, et al. Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs. Lancet. 1992;339:594–8.
   PubMed Web of Science ®Google Scholar
• Ljung R, Petrini P, Lindgren A, Tengborn L, Nilsson IM. Factor VIII and factor IX inhibitors in haemophiliacs. Lancet. 1992;339:1550.
   PubMed Web of Science ®Google Scholar
• Addiego JE-J, Gomperts E, Liu SL, Bailey P, Courter SG, Lee ML, et al. Treatment of hemophilia A with a highly purified factor VIII concentrate prepared by anti-FVIII:C immunoaffinity chromatography. Thromb Haemost. 1992;67:19–27.
   PubMed Web of Science ®Google Scholar
• de Biasi R, Rocino A, Papa E, Salerno E, Mastrullo L, De Blasi D. Incidence of factor VIII inhibitor development in hemophilia A patients treated with less pure plasma derived concentrates. Thromb Haemost. 1994;71:544–7.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• El Alfy MS, Tantawy AA, Ahmed MH, Abdin IA. Frequency of inhibitor development in severe haemophilia A children treated with cryoprecipitate and low-dose immune tolerance induction. Haemophilia. 2000;6:635–8.
   PubMed Web of Science ®Google Scholar
• 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.
   Google Scholar
• Gruppo R, Chen H, Schroth P, Bray GL. Safety and immunogenicity of recombinant factor VIII (Recombinate) in previously untreated patients: a 7.3 year update. Haemophilia. 1998;4:228, ( abstract no. 291).
   Google Scholar
• Goodeve AC, Williams I, Bray GL, Peake IR. Relationship between factor VIII mutation type and inhibitor development in a cohort of previously untreated patients treated with recombinant factor VIII (Recombinate). Recombinate PUP Study Group. Thromb Haemost. 2000;83:844–8.
   PubMed Web of Science ®Google Scholar
• Rothschild C, Laurian Y, Satre EP, Borel Derlon A, Chambost H, Moreau P, 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.
   PubMed Web of Science ®Google Scholar
• 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.
   PubMed Web of Science ®Google Scholar
• Lusher J, Abildgaard C, Arkin S, Mannucci PM, Zimmermann R, Schwartz L, 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.
   PubMed Web of Science ®Google Scholar
• Yoshioka A, Fukutake K, Takamatsu J, Shirahata A, Kogenate Post-Marketing Surveillance Study Group. Evaluation of a recombinant factor VIII preparation (Kogenate) in previously untreated patients with hemophilia A. Int J Hematol. 2003;78:467–74.
   PubMed Web of Science ®Google Scholar
• Giangrande PLF, for the Kogenate Bayer Study Group. Safety and efficacy of Kogenate Bayer in previously untreated patients (PUPs) and minimally treated patients (MTPs). Haemophilia. 2002;8 (suppl. 2):19–22.
   PubMed Web of Science ®Google Scholar
• Kreuz W, Gill JC, Rothschild C, Manco-Johnson MJ, Lusher JM, Kellermann E, 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.
   Web of Science ®Google Scholar
• Oldenburg J, Ivaskevicius V, Schröder J, Müller CR, Ganguly A. Genetic background and inhibitors in previously untreated or minimally treated young patients with severe haemophilia A treated with sucrose-formulated recombinant factor VIII. Thromb Haemost. 2006;95:903–5.
   PubMed Web of Science ®Google Scholar
• Musso R, Santagostino E, Faradji A, et al., Kogenate Bayer European PMS Study Group. Safety and efficacy of sucrose-formulated full-length recombinant factor VIII: experience in the standard clinical setting. Thromb Haemost. 2008;99:52–8.
   PubMed Web of Science ®Google Scholar
• Lusher JM, Lee CA, Kessler CM, Bedrosian CL. The safety and efficacy of B-domain deleted recombinant factor VIII concentrate in patients with severe haemophilia A. Haemophilia. 2003;9:38–49.
   PubMed Web of Science ®Google Scholar
• Lusher JM, Roth DA. The safety and efficacy of B-domain deleted recombinant factor VIII concentrates in patients with severe haemophilia: an update. Haemophilia. 2005;11:292–3.
   PubMed Web of Science ®Google Scholar
• Courter SG, Bedrosian CL. Clinical evaluation of B-domain deleted recombinant factor VIII in previously untreated patients. Semin Hematol. 2001;38:52–9.
   PubMed Web of Science ®Google Scholar
• Pollmann H, Externest D, Ganser A, Eifrig B, Kreuz W, Lenk H, et al. Efficacy, safety and tolerability of recombinant factor VIII (REFACTO) in patients with haemophilia A: interim data from a postmarketing surveillance study in Germany and Austria. Haemophilia. 2007;13:131–43.
   PubMed Web of Science ®Google Scholar