Best evidence on B-domain deletion and the immunogenicity of recombinant factor VIII

Journal of Thrombosis and Haemostasis

Volumn 9, Issue 11, 2011, Pages 2325-2327

  Aledort, L M ^a   Navickis, R J ^b   Wilkes, M M ^b  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^b Hygeia Associates   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALBUMIN; RECOMBINANT BLOOD CLOTTING FACTOR 8;

DRUG EFFICACY; DRUG SAFETY; DRUG SURVEILLANCE PROGRAM; FLUID RESUSCITATION; HEMOPHILIA; HUMAN; IMMUNOGENICITY; LETTER; NONHUMAN; PRIORITY JOURNAL; PROTEIN DOMAIN;

AUTOANTIBODIES; CLINICAL TRIALS AS TOPIC; COAGULANTS; FACTOR VIII; HEMARTHROSIS; HEMOPHILIA A; HOME INFUSION THERAPY; HUMANS; IMMUNOGENETIC PROCESSES; MALE; SECONDARY PREVENTION; SUCROSE;

EID: 80055102357     PISSN: 15387933     EISSN: 15387836     Source Type: Journal    
DOI: 10.1111/j.1538-7836.2011.04496.x     Document Type: Letter

References (37)

1. Aledort LM, Navickis RJ, Wilkes MM. Can B-domain deletion alter the immunogenicity of recombinant factor VIII? J Thromb Haemost 2011; 9: 2180-92.
2. Iorio A, Marcucci M, Makris M. Concentrate related inhibitor risk: is a difference always real? J Thromb Haemost 2011; 9: 2176-9.
3. Schwartz RS, Abildgaard CF, Aledort LM, Arkin S, Bloom AL, Brackmann HH, Brettler DB, Fukui H, Hilgartner MW, Inwood MJ, Kasper CK, Kernoff PBA, Levine PH, Lusher JM, Mannucci PM, Scharrer I, MacKenzie MA, Pancham N, Kuo HS, Allred RU. Human recombinant DNA-derived antihemophilic factor (factor VIII) in the treatment of hemophilia A. The Recombinant Factor VIII Study Group. N Engl J Med 1990; 323: 1800-5.
4. ™) in previously treated patients with hemophilia A. The Recombinate Previously Treated Patient Study Group. Thromb Haemost 1997; 77: 660-7.
5. Seremetis S, Lusher JM, Abildgaard CF, Kasper CK, Allred R, Hurst D. Human recombinant DNA-derived antihaemophilic factor (factor VIII) in the treatment of haemophilia A: conclusions of a 5-year study of home therapy. The Kogenate Study Group. Haemophilia 1999; 5: 9-16.
6. Abshire TC, Brackmann HH, Scharrer I, Hoots K, Gazengel C, Powell JS, Gorina E, Kellermann E, Vosburgh E. Sucrose formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of hemophilia A in home therapy: results of a multicenter, international, clinical investigation. International Kogenate-FS Study Group. Thromb Haemost 2000; 83: 811-6.
7. Courter SG, Bedrosian CL. Clinical evaluation of B-domain deleted recombinant factor VIII in previously treated patients. Semin Hematol 2001; 38: 44-51.
8. Rothschild C, Scharrer I, Brackmann HH, Stieltjes N, Vicariot M, Torchet MF, Effenberger W. European data of a clinical trial with a sucrose formulated recombinant factor VIII in previously treated haemophilia A patients. Haemophilia 2002; 8 (Suppl. 2): 10-4.
9. 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.
10. Tarantino MD, Collins PW, Hay CR, Shapiro AD, Gruppo RA, Berntorp E, Bray GL, Tonetta SA, Schroth PC, Retzios AD, Rogy SS, Sensel MG, Ewenstein BM. Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A. Haemophilia 2004; 10: 428-37.
11. Lusher JM, Roth DA. The safety and efficacy of B-domain deleted recombinant factor VIII concentrates in patients with severe haemophilia A: an update. Haemophilia 2005; 11: 292-3.
12. Shi J, Zhao Y, Wu J, Sun J, Wang L, Yang R. Safety and efficacy of a sucrose-formulated recombinant factor VIII product for the treatment of previously treated patients with haemophilia A in China. Haemophilia 2007; 13: 351-6.
13. Recht M, Nemes L, Matysiak M, Manco-Johnson M, Lusher J, Smith M, Mannucci P, Hay C, Abshire T, O'Brien A, Hayward B, Udata C, Roth DA, Arkin S. Clinical evaluation of moroctocog alfa (AF-CC), a new generation of B-domain deleted recombinant factor VIII (BDDrFVIII) for treatment of haemophilia A: demonstration of safety, efficacy, and pharmacokinetic equivalence to full-length recombinant factor VIII. Haemophilia 2009; 15: 869-80.
14. Smith MP, Giangrande P, Pollman H, Littlewood R, Kollmer C, Feingold J. A postmarketing surveillance study of the safety and efficacy of ReFacto (St Louis-derived active substance) in patients with haemophilia A. Haemophilia 2005; 11: 444-51.
15. ®) in patients with haemophilia A: interim data from a postmarketing surveillance study in Germany and Austria. Haemophilia 2007; 13: 131-43.
16. ® Bayer European PMS Study Group. Thromb Haemost 2008; 99: 52-8.
17. ® utilizing a computer-aided diary: a Nordic multicentre study. Haemophilia 2009; 15: 175-83.
18. Oldenburg J, Goudemand J, Valentino L, Richards M, Luu H, Kriukov A, Gajek H, Spotts G, Ewenstein B. Postauthorization safety surveillance of ADVATE [antihaemophilic factor (recombinant), plasma/albumin-free method] demonstrates efficacy, safety and low-risk for immunogenicity in routine clinical practice. Haemophilia 2010; 16: 866-77.
19. ® in patients with haemophilia A: evaluation of patients' satisfaction after switch to the new reconstitution system. Haemophilia 2010; 16: 66-71.
20. Aygören-Pürsün E, Scharrer I, The German Kogenate Study Group. A multicenter pharmacosurveillance study for the evaluation of the efficacy and safety of recombinant factor VIII in the treatment of patients with hemophilia A. Thromb Haemost 1997; 78: 1352-6.
21. Fukutake K, Musso R, Young J, Norenberg C. Inhibitor formation with sucrose-formulated recombinant factor VIII in patients with hemophilia A: results from postmarketing surveillance studies. J Thromb Haemost 2009; 7 (Suppl. 2): 823.
22. Yoshioka A, Fukutake K, Takamatsu J, Shirahata A. Clinical evaluation of recombinant factor VIII preparation (Kogenate) in previously treated patients with hemophilia A: descriptive meta-analysis of post-marketing study data. Int J Hematol 2006; 84: 158-65.
23. Delumeau JC, Ikegawa C, Yokoyama C, Haupt V. An observational study of sucrose-formulated recombinant factor VIII for Japanese patients with haemophilia A. Thromb Haemost 2008; 100: 32-7.
24. Taki M, Fukutake K, Hanabusa H, Takamatsu J, Shima M, Shirahata A. Post-authorization safety surveillance (PASS) program of antihemophilic factor (recombinant), plasma/albumin-free method (rAHF-PFM) for Japanese hemophilia A patients. J Thromb Haemost 2009; 7 (Suppl. 2): 1125.
25. Gringeri A, Tagliaferri A, Tagariello G, Morfini M, Santagostino E, Mannucci P. Efficacy and inhibitor development in previously treated patients with haemophilia A switched to a B domain-deleted recombinant factor VIII. Br J Haematol 2004; 126: 398-404.
26. Shapiro A, Gruppo R, Pabinger I, Collins PW, Hay CR, Schroth P, Casey K, Patrone L, Ehrlich H, Ewenstein BM. Integrated analysis of safety and efficacy of a plasma- and albumin-free recombinant factor VIII (rAHF-PFM) from six clinical studies in patients with hemophilia A. Expert Opin Biol Ther 2009; 9: 273-83.
27. Young JH, Liu HC, Hsueh EJ, Huang ML, Peng CT, Chen RL, Maas-Enriquez M, Achilles K. Efficacy and safety evaluation of sucrose-formulated recombinant factor VIII for Taiwanese patients with haemophilia A. Haemophilia 2009; 15: 968-70.
28. den Uijl I, Mauser-Bunschoten EP, Roosendaal G, Schutgens R, Fischer K. Efficacy assessment of a new clotting factor concentrate in haemophilia A patients, including prophylactic treatment. Haemophilia 2009; 15: 1215-8.
29. Négrier C, Shapiro A, Berntorp E, Pabinger I, Tarantino M, Retzios A, Schroth P, Ewenstein B. Surgical evaluation of a recombinant factor VIII prepared using a plasma/albumin-free method: efficacy and safety of Advate in previously treated patients. Thromb Haemost 2008; 100: 217-23.
30. Windyga J, Rusen L, Gruppo R, O'Brien AC, Kelly P, Roth DA, Arkin S. BDDrFVIII (Moroctocog alfa [AF-CC]) for surgical haemostasis in patients with haemophilia A: results of a pivotal study. Haemophilia 2010; 16: 731-9.
31. Collins P, Faradji A, Morfini M, Enriquez MM, Schwartz L. Efficacy and safety of secondary prophylactic versus on-demand sucrose-formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13-month crossover study. J Thromb Haemost 2009; 8: 83-9.
32. Auerswald G, Bade A, Johne J, Haubold K, Masurat S, Moorthi C, Overberg D. Prospective study of inhibitor incidence with continuous infusion of factor concentrates during and after surgical procedures in patients with haemophilia A, B, or von Willebrand disease. J Thromb Haemost 2009; 7 (Suppl. 2): 524.
33. Blanchette VS, Shapiro AD, Liesner RJ, Hernández Navarro F, Warrier I, Schroth PC, Spotts G, Ewenstein BM. Plasma and albumin-free recombinant factor VIII: pharmacokinetics, efficacy and safety in previously treated pediatric patients. J Thromb Haemost 2008; 6: 1319-26.
34. Roussel-Robert V, Torchet MF, Legrand F, Rothschild C, Stieltjes N. Factor VIII inhibitors development following introduction of B-domain-deleted recombinant factor VIII in four hemophilia A previously treated patients. J Thromb Haemost 2003; 1: 2450-1.
35. Keeling D. Switching between full-length and B-domain-deleted factor VIII and the risk of inhibitors. Haemophilia 2006; 12: 690-1.
36. Peduzzi P, Concato J, Feinstein AR, Holford TR. Importance of events per independent variable in proportional hazards regression analysis. II. Accuracy and precision of regression estimates. J Clin Epidemiol 1995; 48: 1503-10.
37. Peduzzi P, Concato J, Kemper E, Holford TR, Feinstein AR. A simulation study of the number of events per variable in logistic regression analysis. J Clin Epidemiol 1996; 49: 1373-9.