A multicenter pharmacokinetic study of the B-domain deleted recombinant factor VIII concentrate using different assays and standards

Journal of Thrombosis and Haemostasis

Volumn 1, Issue 11, 2003, Pages 2283-2289

  Morfini, M ^a   Cinotti, S ^a   Bellatrecci A , ^a   Paladino, E ^b   Gringeri, A ^c   Mannucci, P M ^c  

^a University Hospital   (Italy)

^b Medical Department   (Italy)

^c FONDAZIONE IRCCS CA GRANDA OSPEDALE MAGGIORE POLICLINICO   (Italy)

Author keywords

BBD r FVII; Discrepancy; FVII chromogenic one stage assay; Pharmacokinetics

Indexed keywords

B DOMAIN DELETED FACTOR VIII; B-DOMAIN-DELETED FACTOR VIII; BLOOD CLOTTING FACTOR 8; CHROMOGENIC SUBSTRATE; DIAGNOSTIC AGENT; PEPTIDE FRAGMENT;

ADULT; ARTICLE; BLOOD; BLOOD CLOTTING TEST; CLINICAL TRIAL; COMPARATIVE STUDY; HALF LIFE TIME; HEMOPHILIA A; HUMAN; METHODOLOGY; MULTICENTER STUDY; PHARMACOKINETICS; STANDARD; TECHNIQUE;

ADULT; BLOOD COAGULATION TESTS; CHROMOGENIC COMPOUNDS; FACTOR VIII; HALF-LIFE; HEMOPHILIA A; HUMANS; METHODS; PEPTIDE FRAGMENTS; PHARMACOKINETICS; REFERENCE STANDARDS;

EID: 2542605872     PISSN: 15387933     EISSN: 15387836     Source Type: Journal    
DOI: 10.1046/j.1538-7836.2003.00481.x     Document Type: Article

References (36)

1. Pittman DD, Alderman EM, Tomkinson KN, Wang JH, Giles AR, Kaufman RJ. Biochemical, immunological, and in vivo functional char acterization of B-domain-deleted factor VIII. Blood 1993; 81: 2925-35.
2. Lenting PH, van Mourick JA, Mertens K. The life cycle of coagulation factor VIII in view of its structure and function. Blood 1998; 92: 3983-96.
3. Pittman DD, Marquette KA, Kaufman RJ. Role of B domain for factor VIII and factor V expression and function. Blood 1994; 84: 4214-25.
4. Eriksson RK, Fenge C, Lindner-Olsson E, Ljungqvist C, Rosenquist J, Smeds AL, Ostlin A, Charlebois T, Leonard M, Kelley BD, Ljungqvist A. The manufacturing process for B-domain deleted recombinant Factor VIII. Semin Hematol 2001; 38 (S4): 24-31.
5. Osterberg T, Fatouros E, Neidhardt E, Warne N, Mikaelsson M. B- domain deleted recombinant factor VIII formulation and stability. Semin Hematol 2001; 38 (S4): 40-3.
6. Sandberg H, Almsted A, Brandt J, Castro VM, Gray E, Holmquist L, Lewin M, Oswaldsson U, Mikaelsson M, Jankowski MA, Bond M, Scoble HA. Structural and functional characterization of B-domain deleted recombinant Factor VIII. Semin Hematol 2001; 38 (S4): 4-12.
7. Mikaelsson M, Oswaldsson U, Sandberg H. Influence of phospholipids on the assessment of factor VIII activity. Haemophilia 1998; 4: 646-50.
8. Mikaelsson M, Oswaldsson U, Frank L. Comparison of activity and antigen measurements in a pharmacokinetic study of recombinant factor VIII SQ. Haemophilia 1998; 4: 187(Abstract).
9. Oswaldsson U, Mikaelsson M. Comparison of factor VIII: C methods for potency assessment of factor VIII concentrates. Haemophilia 1998; 4: 187(Abstract).
10. Kirkwood TBL, Barrowcliffe TW. Discrepancies between one-stage and two-stage assay of Factor VIII. C Br J Haematol 1978; 40: 333-8.
11. Barrowcliffe TW. Recommendations for the assay of high-purity Factor VIII concentrates. Thromb Haemost 1993; 70: 876-7.
12. Barrowcliffe TW. Standardization and assay. Semin Thromb Hemost 1993; 19: 73-9.
13. Barrowcliffe TW, Raut S, Hubbard R. Discrepancies in potency assess ment of recombinant FVIII concentrates. Haemophilia 1998; 4: 634-40.
14. Barrowcliffe TW, Raut S, Sands D, Hubbard A. Coagulation and chromogenic assays of Factor VIII activity: general aspects, standardi zation and recommendations. Semin Thromb Hemost 2002; 28: 247-55.
15. Hubbard AR, Bevan SA, Weller LJ. Potency estimation of recombinant factor VIII: effect of assay method and standard. Br J Haematol 2001; 113: 533-6.
16. Oswaldsson U, Frank L, Mikaelsson M. Comparison of normal plasma and product-specific standards in the assessment of post-injection factor VIII activity. Thromb Haemost 1999; 574 (Suppl.): (Abstract).
17. Ingerslev J, Oswaldsson U, Renlund S, Mikaelsson M and the Nordic ReFacto Study Group. Results of a collaborative Nordic field Study (RASPUTIN) on determination of recombinant B-domain deleted Factor VIII in plasma employing a (Refacto) concentrate standard. Thromb Haemost 2001; Suppl.: (Abstract).
18. Katsarou O, Anastasopoulou J, Kotsi P, Xiarhopoulou P, Loukopoulou E, Montor B, Karafoulidou A. Refacto experience: clinical use and pharmacokinetic evaluation using one-stage clotting and chromogenic Assays. Thromb Haemost 2001; Suppl.: (Abstract).
19. Morfini M, Lee M, Messori A. The design and analysis of half-life and recovery studies for factor VIII and factor IX. Thromb Haemost 1991; 66: 384-6.
20. Ingram GI. Blood-coagulation factor VIII: genetics, physiological control, and bioassay. Adv Clin Chem 1965; 8: 189-236.
21. Messori A, Bosi A, Guidi S, Longo G, Pistolesi C, Saccardi R, Valenza T, Vannucchi AM. PKRD: a pharmacokinetic program for least-squares and Bayesan analysis of repeated-dose pharmacokinetic curves. Comp Meth Progr Biomed 1992; 38: 27-35.
22. Nilsson IM, Hedner U. Characteristics of various factor VIII concentrates used in treatment of haemophilia A. Br J Haematol 1977; 37: 543-57.
23. Prowse CV In vivo recovery of factor VIII following transfusion: a survey of recent data and publications to assess the influence of standards used for potency assignment. Thromb Haemost 1995; 74: 1191-6.
24. Messori A, Longo G, Morfini M, Cinotti S, Filimberti E, Giustarini G, Rossi Ferrini P. Multi-variate analysis of factors governing the pharmacokinetics of exogenous factor VIII in haemophiliacs. Eur J Clin Pharmacol 1988; 35: 663-8.
25. Barrowcliffe TW, Watton J, Harmon A, Tubbs JF, Kemball-Cook G. Potency of high purity factor VIII concentrates. Lancet 1990; ii: 124.
26. Kemball-Cook G, Tubbs JE, Dawson NJ, Barrowcliffe TW. The beha vior of different factor VIII concentrates in a chromogenic factor X- activating system. Br J Haematol 1993; 84: 273-8.
27. Niemetz J, Nossel HL Activated coagulation factors: in vivo and in vitro studies. Br J Haematol 1969; 16: 337-51.
28. Mikaelsson M, Oswaldsson U, Nilsson IM. Potency and in vivo recovery of high purity factor VIII concentrates. Thromb Haemost 1994; 72: 160-1.
29. Lusher JM, Hillman-Wiseman C, Hurst D. In vivo recovery with products of very high purity-assay discrepancies. Haemophilia 1998; 4: 641-5.
30. Frank L, Karstrom K, Liden Y, Mansouri M, Sigle J, Oswaldsson U. Comparison of factor VIII methods in the assessment of post injection patient plasma samples. Haemophilia 1998; 4: 187(Abstract).
31. Mikaelsson M, Oswaldsson U. Assaying the circulating factor VIII activity in hemophilia A patients treated with recombinant factor VIII products. Semin Thromb Hemost 2002; 28: 257-64.
32. Lee C, Owens D, Bray G, Giangrande P, Collins P, Hay C, Gomperts E, Schroth P, Barrowcliffe T. Pharmacokinetics of recombinant factor VIIII (Recombinate) using one-stage clotting and chromogenic factor VIII assay. Thromb Haemost 1999; 82: 1644-7.
33. Lee C, Barrowcliffe T, Gomperts E, Hubbard A, Kemball-Cook G, Owens D, Von Tilberg L, Pasi J. Pharmacokinetic in vivo comparison using 1-stage and chromogenic substrate assays with two formulation of Hemofil-M. Thromb Haemost 1996; 76: 950-6.
34. Lee C. The use of recombinant Factor VIII products in previously treated patients with hemophilia A: pharmacokinetics, efficacy, safety and inhibitor development. Semin Thromb Haemost 2002; 28: 241-6.
35. Mikaelsson M, Oswaldsson U, Jankowski MA. Measurement of Factor VIII activity of B-domain deleted recombinant factor VIII. Semin Hematol 2001; 38 (S4): 13-23.
36. Fijnvandraat K, Berntorp E, ten Cate JW, Johnsson H, Peters M, Savidge G, Tengborn L, Spira J, Stahl C. Recombinant B-domain deleted Factor VIII (r-VIII SQ): pharmacokinetics and initial safety aspects in hemo philia A patients. Thromb Haemost 1997; 77: 298-302.