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

Volumn 16, Issue 6, 2010, Pages 866-877

  Oldenburg, Johannes ^a   Goudemand, J ^b   Valentino, L ^c   Richards, M ^d   Luu, H ^e   Kriukov, A ^e   Gajek, H ^e   Spotts, G ^e   Ewenstein, B ^e  

^a UNIVERSITY HOSPITAL BONN   (Germany)

^b UNIV LILLE   (France)

^c RUSH UNIVERSITY MEDICAL CENTER   (United States)

^d ST JAMES S UNIVERSITY HOSPITAL   (United Kingdom)

^e BAXTER HEALTHCARE CORPORATION   (United States)

Author keywords

Factor VIII; Haemophilia A; Inhibitor; Postmarketing; RAHF; RAHF PFM; RFVIII; Safety; Surveillance; Third generation

Indexed keywords

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

ABDOMINAL PAIN; ADOLESCENT; ARTICLE; ASTHENIA; BLEEDING; CHILD; CLINICAL PRACTICE; DRUG EFFICACY; DRUG HYPERSENSITIVITY; DRUG SAFETY; DRUG SURVEILLANCE PROGRAM; DRUG WITHDRAWAL; FATIGUE; FEMALE; HEADACHE; HEMOPHILIA A; HUMAN; IMMUNOGENICITY; MAJOR CLINICAL STUDY; MALE; PRIORITY JOURNAL; PROPHYLAXIS; PSYCHOMOTOR ACTIVITY; SCHOOL CHILD; SIDE EFFECT; SKIN ODOR; URINE ODOR;

ADOLESCENT; ADULT; BLOOD COAGULATION FACTOR INHIBITORS; CHILD; CHILD, PRESCHOOL; FACTOR VIII; FEMALE; HEMOPHILIA A; HEMORRHAGE; HUMANS; MALE; MIDDLE AGED; PRODUCT SURVEILLANCE, POSTMARKETING; RECOMBINANT PROTEINS; UNITED STATES;

EID: 78049307974     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2010.02332.x     Document Type: Article

References (54)

1. Clark DW, Harrison-Woolrych M. The role of the New Zealand Intensive Medicines Monitoring Programme in identification of previously unrecognised signals of adverse drug reactions. Curr Drug Saf 2006; 1: 169-78.
2. Mehran R, Leon MB, Feigal DA et al. Post-market approval surveillance: a call for a more integrated and comprehensive approach. Circulation 2004; 109: 3073-7.
3. Roeser HP, Rohan AP. Post-marketing surveillance of drugs. The spontaneous reporting scheme: role of the Adverse Drug Reactions Advisory Committee. Med J Aust 1990; 153: 720-6.
4. Westerholm B. The rationale for a post-marketing surveillance. Hum Reprod 1987; 2: 41-4.
5. Guidance for Industry. Reports on the Status of Postmarketing Study Commitments - Implementation of Section 130 of the Food and Drug Administration Modernization Act of 1997. U.S. Department of Health and Human Services Food and Drug Administration Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER) 2006. Available at .Accessed May 28, 2010.
6. Key NS. Inhibitors in congenital coagulation disorders. Br J Haematol 2004; 127: 379-91.
7. Kreuz W, Becker S, Lenz E et al. Factor VIII inhibitors in patients with hemophilia A: epidemiology of inhibitor development and induction of immune tolerance for factor VIII. Semin Thromb Hemost 1995; 21: 382-9.
8. Lusher J, Abildgaard C, Arkin S 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.
9. Lassila R, Rothschild C, De MP, Richards M, Perez R, Gajek H. Recommendations for postmarketing surveillance studies in haemophilia and other bleeding disorders. Haemophilia 2005; 11: 353-9.
10. Neugebauer B, Drai C, Haase M et al. Factor VIII products and inhibitor development: concepts for revision of European regulatory guidelines. Haemophilia 2008; 14: 142-4.
11. Cahill MR, Colvin BT. Haemophilia. Postgrad Med J 1997; 73: 201-6.
12. Fricke WA, Lamb MA. Viral safety of clotting factor concentrates. Semin Thromb Hemost 1993; 19: 54-61.
13. Julius C, Westphal RG. The safety of blood components and derivatives. Hematol Oncol Clin North Am 1992; 6: 1057-77.
14. Lee CA. The natural history of HIV disease in haemophilia. Blood Rev 1998; 12: 135-44.
15. Gerritzen A, Schneweis KE, Brackmann HH et al. Acute hepatitis A in haemophiliacs. Lancet 1992; 340: 1231-2.
16. Kleim JP, Bailly E, Schneweis KE et al. Acute HIV-1 infection in patients with hemophilia B treated with beta-propiolactone-UV-inactivated clotting factor. Thromb Haemost 1990; 64: 336-7.
17. Ewenstein BM, Collins P, Tarantino MD et al. Hemophilia therapy innovation: development of an advanced category recombinant factor VIII by a plasma/albumin-free method. Proceedings of a Special Symposium at the XIXth Congress of the International Society on Thrombosis and Haemostasis, July 12-18, 2003, Birmingham, UK. Semin Hematol 2004; 41(1 Suppl. 2): 1-16.
18. Farrugia A. Evolving perspectives in product safety for haemophilia. Haemophilia 2002; 8: 236-43.
19. Kasper CK. Concentrate safety and efficacy. Haemophilia 2002; 8: 161-5.
20. Lee DC, Miller JL, Petteway SR Jr. Pathogen safety of manufacturing processes for biological products: special emphasis on KOGENATE Bayer. Haemophilia 2002; 8(Suppl. 2): 6-9.
21. Medical and Scientific Advisory Council (MASAC). Recommendations Concerning the Treatment of Hemophilia and other Bleeding Disorders (#187). MASAC Document 2008Nov 16 2008(no. 187). Available at Accessed February 18, 2010.
22. Dunn AL, Abshire TC. Current issues in prophylactic therapy for persons with hemophilia. Acta Haematol 2006; 115: 162-71.
23. EMEA. Report of Expert Meeting On Factor VIII Products and Inhibitor Development, 28 February 2006 - 2 March 2006. London, 22 February 2007 2007Doc. Ref: EMEA/CHMP/BPWP/123835/2006.
24. Oldenburg J, Pavlova A. Genetic risk factors for inhibitors to factors VIII and IX. Haemophilia 2006; 12(Suppl. 6): 15-22.
25. Hay CR. The epidemiology of factor VIII inhibitors. Haemophilia 2006; 12(Suppl. 6): 23-8.
26. Peerlinck K, Arnout J, Gilles JG, Saint-Remy JM, Vermylen J. A higher than expected incidence of factor VIII inhibitors in multitransfused haemophilia A patients treated with an intermediate purity pasteurized factor VIII concentrate. Thromb Haemost 1993; 69: 115-8.
27. Rosendaal FR, Nieuwenhuis HK, Van Den Berg HM et al. A sudden increase in factor VIII inhibitor development in multitransfused hemophilia A patients in The Netherlands. Dutch Hemophilia Study Group. Blood 1993; 81: 2180-6.
28. Blanchette VS, Shapiro AD, Liesner RJ et al. Plasma and albumin-free recombinant factor VIII: pharmacokinetics, efficacy and safety in previously treated pediatric patients. J Thromb Haemost 2008; 6: 1319-26.
29. Negrier C, Shapiro A, Berntorp E et al. 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. Tarantino MD, Collins PW, Hay CR et al. 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.
31. Shapiro A, Gruppo R, Pabinger I et al. 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.
32. Shapiro AD. Anti-hemophilic factor (recombinant), plasma/albumin-free method (octocog-alpha; ADVATE) in the management of hemophilia A. Vasc Health Risk Manag 2007; 3: 555-65.
33. Suissa S, Garbe E. Primer: administrative health databases in observational studies of drug effects - advantages and disadvantages. Nat Clin Pract Rheumatol 2007; 3: 725-32.
34. Walker I, Pai M, Akabutu J et al. The Canadian Hemophilia Registry as the basis for a national system for monitoring the use of factor concentrates. Transfusion 1995; 35: 548-51.
35. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
36. Lusher JM. First and second generation recombinant factor VIII concentrates in previously untreated patients: recovery, safety, efficacy, and inhibitor development. Semin Thromb Hemost 2002; 28: 273-6.
37. 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.
38. Giles AR, Rivard GE, Teitel J, Walker I. Surveillance for factor VIII inhibitor development in the Canadian Hemophilia A population following the widespread introduction of recombinant factor VIII replacement therapy. Transfus Sci 1998; 19: 139-48.
39. 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; 93: 457-67.
40. Scharrer I. Experience with KOGENATE Bayer in surgical procedures. Haemophilia 2002; 8(Suppl. 2): 15-8.
41. 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.
42. Dimichele DM, Hay CR. The international immune tolerance study: a multicenter prospective randomized trial in progress. J Thromb Haemost 2006; 4: 2271-3.
43. Ghosh K, Jijina F, Shetty S, Madkaikar M, Mohanty D. First-time development of FVIII inhibitor in haemophilia patients during the postoperative period. Haemophilia 2002; 8: 776-80.
44. Sharathkumar A, Lillicrap D, Blanchette VS et al. Intensive exposure to factor VIII is a risk factor for inhibitor development in mild hemophilia A. J Thromb Haemost 2003; 1: 1228-36.
45. White B, Cotter M, Byrne M, O'Shea E, Smith OP. High responding factor VIII inhibitors in mild haemophilia - is there a link with recent changes in clinical practice? Haemophilia 2000; 6: 113-5.
46. Singleton E, Smith J, Kavanagh M, Nolan B, White B. Low risk of inhibitor formation in haemophilia patients after a change in treatment from Chinese hamster ovary cell-produced to baby hamster kidney cell-produced recombinant factor VIII. Thromb Haemost 2007; 98: 1188-92.
47. 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.
48. White GC, DiMichele D, Mertens K et al. Utilization of previously treated patients (PTPs), noninfected patients (NIPs), and previously untreated patients (PUPs) in the evaluation of new factor VIII and factor IX concentrates. Recommendation of the Scientific Subcommittee on Factor VIII and Factor IX of the Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost 1999; 81: 462.
49. Kempton CL, Soucie JM, Abshire TC. Incidence of inhibitors in a cohort of 838 males with hemophilia A previously treated with factor VIII concentrates. J Thromb Haemost 2006; 4: 2576-81.
50. Abshire TC, Brackmann HH, Scharrer I et al. Sucrose formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of hemophilia A in home therapy - International Kogenate-FS Study Group. Thromb Haemost 2000; 83: 811-6.
51. Courter SG, Bedrosian CL. Clinical evaluation of B-domain deleted recombinant factor VIII in previously treated patients. Semin Hematol 2001; 38(2 Suppl. 4): 44-51.
52. Schwartz RS, Abildgaard CF, Aledort LM et al. Human recombinant DNA-derived antihemophilic factor (factor VIII) in the treatment of hemophilia A. recombinant Factor VIII Study Group. N Engl J Med 1990; 323: 1800-5.
53. White GC, Courter S, Bray GL, Lee M, Gomperts ED. A multicenter study of recombinant factor VIII (Recombinate) in previously treated patients with hemophilia A. The Recombinate Previously Treated Patient Study Group. Thromb Haemost 1997; 77: 660-7.
54. NG T-H. Sample Size Determination in the Safety Evaluation of FVIII Products. Bethesda, MD: CBER Workshop on Inhibitors to Factor VIII. Available at Accessed November 21, 2003.