The need for previously untreated patient population studies in understanding the development of factor VIII inhibitors

Haemophilia

Volumn 12, Issue 6, 2006, Pages 573-578

  Gomperts, E D ^a  

^a UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

Clinical studies; Factor VIII; Haemophilia A; Inhibitors; Previously untreated patients; Risk factors

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; BLOOD CLOTTING FACTOR 8 CONCENTRATE; DRUG ANTIBODY; RECOMBINANT BLOOD CLOTTING FACTOR 8; VON WILLEBRAND FACTOR;

ANTIBODY PRODUCTION; ANTIBODY TITER; BLEEDING; CLINICAL TRIAL; COHORT ANALYSIS; CONTINUOUS INFUSION; DISEASE SEVERITY; DRUG EFFICACY; DRUG SYNTHESIS; GENE MUTATION; GENETIC ASSOCIATION; GENETIC CODE; GENETIC RISK; GENETIC SCREENING; GENETIC SUSCEPTIBILITY; HEMOPHILIA A; HUMAN; PHENOTYPE; PRIORITY JOURNAL; REVIEW; RISK ASSESSMENT; RISK FACTOR; TREATMENT DURATION;

BLOOD COAGULATION FACTOR INHIBITORS; FACTOR VIII; HEMOPHILIA A; HUMANS; RISK FACTORS;

EID: 33750704965     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2006.01341.x     Document Type: Review

References (37)

1. DiMichele D, Rivard G, Hay C, Antunes S. Inhibitors in haemophilia: clinical aspects. Haemophilia 2004; 10 (Suppl. 4): 140-5.
2. White GC, DiMichele DM, 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. Thromb Haemost 1999; 81: 462.
3. The European Agency for the Evaluation of Medicinal Products. Note for Guidance on the Clinical Investigation of Recombinant Factor VIII and IX Products, 2000, CPMP/BPWP/1561/99.
4. DiMichele D. Inhibitors: Resolving diagnostic and therapeutic dilemmas. Haemophilia 2002; 8: 280-7.
5. Lusher JM, Salzman PM, Monoclate Study Group. Viral safety and inhibitor development associated with factor VIIIC ultra-purified from plasma in hemophiliacs previously unexposed to factor VIIIC concentrates. Semin Hematol 1990; 27 (Suppl. 2): 1-7.
6. Addiego JE, Gomperts E, Liu SL et.al. Treatment of hemophilia A with a highly purified factor VIII concentrate prepared by anti-FVIIIc immunoaffinity chromatography. Thromb Haemost 1992; 67: 19-27.
7. Bray GL, Gomperts ED, Courter S et.al. A multicenter study of recombinant factor VIII (Recombinate TM): Safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. Blood 1994; 83: 2428-35.
8. Yee TT, Williams MD, Hill FGH et.al. 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.
9. Courter SG, Bedrosian CL. Clinical evaluation of B-domain deleted recombinant factor VIII in previously untreated patients. Semin Hematol 2001; 38 (Suppl. 4): 52-9.
10. 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.
11. Kruez 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. Thromb Haemost 2005; 93: 457-67.
12. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: A systematic review. Haemophilia 2003; 9: 418-35.
13. Gill FM. The natural history of factor VIII inhibitors in patients with hemophilia A. Prog Clin Biol Res 1984; 150: 19-29.
14. Addiego JE Jr, Kasper C, Abildgaard C et.al. Increased frequency of inhibitors in African American hemophilia A patients. Blood 1994; 1 (Suppl.): 239a.
15. Schwaab R, Brackmann HH, Meyer C et.al. Haemophilia A: Mutation type determines risk of inhibitor formation. Thromb Haemost 1995; 74: 1402-6.
16. Oldenburg J, Picard JK, Schwaab R et.al. HLA genotype of patients with severe haemophilia A due to intron 22 inversion with and without inhibitors of factor VIII. Thromb Haemost 1997; 77: 238-42.
17. Astermark J, Berntorp E, White GC et.al. The MalmöInternational Brother Study (MIBS): Further support for genetic predisposition to inhibitor development. Haemophilia 2001; 7: 267-72.
18. Astermark J, Oldenbrug O, Pavlova A et.al. 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.
19. Berntorp E, Astermark J, Donfield SM et.al. Haemophilia Inhibitor Genetics Study - evaluation of a model for studies of complex diseases using linkage and association methods. Haemophilia 2005; 11: 427-9.
20. 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.
21. Morado M, Villar A, Jimenez Yuste V et.al. Prophylactic treatment effects on inhibitor risk: Experience in one centre. Haemophilia 2005; 11: 79-83.
22. 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.
23. van der Bom JG, Mauser-Bunschoten EP, Fischer K, van den Berg HM. Age of first treatment and immune tolerance to factor VIII in severe hemophilia. Thromb Haemost 2003;89: 475-9.
24. Gouw SC. The concerted action on neutralizing antibodies in severe haemophilia A (Canal Study): Preliminary findings. J Thromb Haemost 2005; 3 (Suppl. 1): OR093.
25. von Auer C, Oldenburg J, Auerswald G et.al. The development of inhibitors direct against factor VIII after continuous infusion of factor VIII concentrates in patients with hemophilia A. J Thromb Haemost 2004; 1 (Suppl. 1): P1623 (Abstract).
26. Sharathkumar A, Lillicrap D, Blanchette VS et.al. Intensive exposure to factor VIII is a risk for inhibitor development in mild hemophilia A. J Thromb Haemost 2003; 1: 1228-36.
27. Ghosh K, Jijina F, Shetty S et.al. First-time development of FVIII inhibitor in haemophilia patients during the postoperative period. Haemophilia 2002; 8: 776-80.
28. Goudemand J, Rothschild C, Demiguel V 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.
29. Scharrer I, Bray GL, Neutzling O. Incidence of inhibitors in haemophilia A patients -a review of recent studies of recombinant and plasma-derived factor VIII concentrates. Haemophilia 1999; 5: 145-54.
30. Rivard GE, Lillicrap D, Poon MC et.al. Can activated recombinant factor VII be use to postpone the exposure of infants to factor VIII until after 2years of age? Haemophilia 2005; 11: 335-9.
31. Goodeve AC, Williams I, Bray GL et.al. Relationship between factor VIII mutation type and inhibitor development in a cohort of previously untreated patients treated with recombinant factor VIII (Recombinate ®). Thromb Haemost 2000; 83: 844-8.
32. Kruez W, Escuriola-Ettingshausen E, Auerswald G et.al. Epidemiology of inhibitors and current treatment strategies. Haematologica 2003; 88: EREP04.
33. Ehrenforth S, Kreuz W, Scharrer I et.al. Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs. Lancet 1992; 339: 594-8.
34. de Biasi R, Rocino A, Papa E et.al. Incidence of factor VIII inhibitor development in hemophilia A patients treated with less pure plasma derived concentrates. Thromb Haemost 1994; 71: 544-7.
35. Addiego J, Kasper C, Abildgaard C et.al. Frequency of inhibitor development in haemophiliacs treated with low-purity factor VIII. Lancet 1993; 342: 462-4.
36. Mannucci PM. Assessing the risk of inhibitor formation with different factor VIII products. Blood 2006; 107; 3809-10.
37. Donadel-Claeyssens S. Current co-ordinated activities of the PEDNET (European Paediatric Network for Haemophilia Management). Haemophila 2006; 12; 124-7.