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

Thrombosis and Haemostasis

Volumn 93, Issue 3, 2005, Pages 457-467

  Kreuz, Wolfhart ^g   Gill, Joan C ^a   Rothschild, Chantal ^b   Manco Johnson, Marilyn J ^c   Lusher, Jeanne M ^d   Kellermann, Elke ^e   Gorina, Eduard ^f   Larson, Peter J ^f  

^a MEDICAL COLLEGE OF WISCONSIN   (United States)

^b HÔPITAL NECKER ENFANTS MALADES   (France)

^c UNIVERSITY OF COLORADO   (United States)

^d CHILDREN'S HOSPITAL OF MICHIGAN   (United States)

^e BAYER AG   (Germany)

^f BAYER CORPORATION   (United States)

^g UNIVERSITY HOSPITAL FRANKFURT   (Germany)

Author keywords

FVIII inhibitor; Haemophilia A; Pediatric; PUPs

Indexed keywords

BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 8 INHIBITOR; NEUTRALIZING ANTIBODY; RECOMBINANT BLOOD CLOTTING FACTOR 8; SUCROSE;

ANTIBODY PRODUCTION; ARTICLE; BLEEDING; CATHETERIZATION; CHILD CARE; CONSTIPATION; CONTROLLED STUDY; DISEASE SEVERITY; DOSE RESPONSE; DRUG EFFICACY; DRUG HYPERSENSITIVITY; DRUG SAFETY; GENE MUTATION; HEMOPHILIA A; HUMAN; INFANT; MAJOR CLINICAL STUDY; PRESCHOOL CHILD; PRIORITY JOURNAL; PROPHYLAXIS; RESPIRATORY TRACT INFECTION; SEROCONVERSION; SURGICAL TECHNIQUE; TREATMENT OUTCOME; VEIN PUNCTURE;

ANTIBODY FORMATION; CHILD, PRESCHOOL; DNA MUTATIONAL ANALYSIS; EXONS; FACTOR VIII; HEMOPHILIA A; HEMORRHAGE; HUMANS; INFANT; INTRONS; MUTATION; TREATMENT OUTCOME;

EID: 15344351616     PISSN: 03406245     EISSN: None     Source Type: Journal    
DOI: 10.1160/TH03-10-0643     Document Type: Article

References (32)

1. Giles AR, Rivard GE, Teitel J, et al. Surveillance for factor VIII inhibitor development in the Canadian Haemophilia A population following the widespread introduction of recombinant factor VIII replacement therapy. Transfus Sci 1998; 19: 139-48.
2. 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 haemophilia A. Thromb Haemost 1997; 78: 1352-6.
3. Seremetis S, Lusher JM, Abildgaard CF, et al. Human recombinant DNA-derived antihaemophilic factor (factor VIII) in the treatment of haemophilia A: conclusions of a 5-year study of home therapy. Haemophilia 1999; 5: 9-16.
4. Lusher JM, Arkin S, Abildgaard CF, et al. Kogenate treatment of previously untreated patients (PUPs) with haemophilia A: update of safety, efficacy, and inhibitor development after 7 study years. Blood 1996; 88(10): 442 A (Abstract 1755).
5. Medical and Scientific Advisory Council (MASAC). Recommendation regarding the use of recombinant factor VIII in the treatment of haemophilia A. National Haemophilia Foundation 1995; Medical Bulletin # 232 (Chapter Advisory # 234).
6. Boedeker BG. Production processes of licensed recombinant factor VIII preparations. Semin Thromb Hemost 2001; 27: 385-94.
7. Brackmann HH, Abshire T, Scharrer I, et al. Safety and efficacy of a sucrose-formulated recombinant factor VIII (rFVIII-SF) during home treatment of patients with severe haemophilia A. Haemophilia 1998; 4: 190.
8. Abshire T, Brackmann H-H, Scharrer I, et al. Sucrose-formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of haemophilia A in home therapy: results of a multicenter, international, clinical investigation. Thromb Haemost 2000; 83: 811-16.
9. Scharrer I, Brackmann H-H, Sultan Y, et al. Efficacy of a sucrose formulated recombinant factor VIII (rFVIII-SF) used for 22 surgical procedures in patients suffering from severe haemophilia A. Haemophilia 2000; 6: 614-18.
10. The European Agency for the Evaluation of Medicinal Products. Note for guidance to assess efficacy and safety of human plasma derived factor VIII: C and factor IX: C products in clinical trials in haemophiliacs before and after authorisation. 1996: CPMP/198/95 Final.
11. 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.
12. 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.
13. Verbruggen B, Novakova I, Wessels H, et al. The Nijmegen modification of the Bethesda assay for factor VIII:C inhibitors: improved specificity and reliability. Thromb Haemost 1995; 73: 247-51.
14. Larson PJ, Zhang C, Gorina E, et al. IgG formation to mammalian proteins in haemophilia patients following treatment with a new recombinant human factor VIII. Blood 2002; 100(11 (Part 2 of 2 Parts)): 110b.
15. 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.
16. Bray GL, Gomperts ED, Courter S, et al. A multicenter study of recombinant factor VIII (Recombinate): safety, efficacy, and inhibitor risk in previously untreated patients with haemophilia A. Blood 1994; 83: 2428-35.
17. Hay CR. Factor VIII inhibitors in mild and moderate-severity haemophilia A. Haemophilia 1998; 4: 558-63.
18. Ohta H, Takahashi I, Kojima T, et al. Histocompatibility antigens and alleles in Japanese haemophilia A patients with or without factor VIII antibodies. Tissue Antigens 1999; 54: 91-7.
19. Oldenburg J, Brackmann HH, Schwaab R. Risk factors for inhibitor development in haemophilia A. Haematologica 2000; 85(10 Suppl): 7-13; discussion 13-4.
20. Astermark J, Berntorp E, White GC, et al. The Malmo International Brother Study (MIBS): further support for genetic predisposition to inhibitor development in haemophilia patients. Haemophilia 2001; 7: 267-72.
21. Schwaab R, Brackmann HH, Meyer C, et al. Haemophilia A: mutation type determines risk of inhibitor formation. Thromb Haemost 1995; 74: 1402-6.
22. Millar DS, Steinbrecher RA, Wieland K, et al. The molecular genetic analysis of haemophilia A; characterization of six partial deletions in the factor VIII gene. Hum Genet 1990; 86: 219-27.
23. Lorenzo JI, Lopez A, Altisent C, et al. Incidence of factor VIII inhibitors in severe haemophilia: the importance of patient age. Br J Haematol 2001; 113: 600-3.
24. Van Der Bom JG, Mauser-Bunschoten EP, Fischer K, et al. Age at first treatment and immune tolerance to factor VIII in severe haemophilia. Thromb Haemost 2003; 89: 475-9.
25. 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.
26. 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.
27. Lusher J, Abildgaard C, Arkin S, et al. Human recombinant DNA-derived antihemophilic factor in the treatment of previously untreated patients with haemophilia A: final report on a hallmark clinical investigation. J Thromb Haemost 2004; 2: 574-83.
28. Yoshioka A, Shima M, Fakutake K, et al. and the KOGENATE® FS Study Group. Safety and efficacy of a new recombinant FVIII formulated with sucrose (rFVIII-FS) in patients with haemophilia A: a long-term, multi-centre clinical study in Japan. Haemophilia 2001; 7: 242-49.
29. Larson P, Zhang C, Gorina E, et al. IgG formation to mammalian proteins in haemophilia A patients following treatment with a new recombinant human factor VIII. J Thromb Haemost 2004; 2: 1011-12.
30. Ingerslev J, Christiansen K, Ravn HB, et al. Antibodies to heterologous proteins in haemophilia A patients receiving recombinant factor VIII (Recombinate). Thromb Haemost 2002; 87: 626-34.
31. Yoshioka A, Fukutake K, Takamatsu J, et al. and the Kogenate Post-Marketing Surveillance Study Group. Clinical evaluation of recombinant factor VIII preparation (Kogenate) in previously untreated patients with haemophilia A. Int J Hematol 2003; 78: 467-74.
32. Suiter TM. First and next generation native rFVIII in the treatment of haemophilia A. What has been achieved? Can patients be switched safety? Semin Thromb Hemost 2002; 28: 277-84.