Time to think outside the box? Proposals for a new approach to future pharmacokinetic studies of von Willebrand factor concentrates in people with von Willebrand disease

Seminars in Thrombosis and Hemostasis

Volumn 33, Issue 8, 2007, Pages 745-758

  Favaloro, Emmanuel J ^a   Kershaw, Geoff ^b   McLachlan, Andrew J ^c   Lloyd, John ^d  

^a WESTMEAD HOSPITAL   (Australia)

^b ROYAL PRINCE ALFRED HOSPITAL   (Australia)

^c UNIVERSITY OF SYDNEY   (Australia)

^d INSTITUTE OF MEDICAL AND VETERINARY SCIENCE   (Australia)

Author keywords

Coagulation factor concentratepharmacokinetics; Von Willebrand disease; Von Willebrand factor; VWD; VWF

Indexed keywords

BLOOD CLOTTING FACTOR 8; RISTOCETIN; VON WILLEBRAND FACTOR;

BINDING ASSAY; DENSITOMETRY; DRUG HALF LIFE; ENZYME BINDING; EVALUATION; MOLECULAR WEIGHT; PRIORITY JOURNAL; REVIEW; VON WILLEBRAND DISEASE;

COLLAGEN; FACTOR VIII; HUMANS; RISTOCETIN; VON WILLEBRAND DISEASE; VON WILLEBRAND FACTOR;

EID: 38549152286     PISSN: 00946176     EISSN: None     Source Type: Journal    
DOI: 10.1055/s-2007-1000367     Document Type: Review

References (35)

1. Sadler JE, Budde U, Eikenboom JCJ, et al. Update on the pathophysiology and classification of von Willebrand disease: a report of the Subcommittee on von Willebrand Factor. J Thromb Haemost 2006;4:2103-2114
2. Federici AB. Management of von Willebrand disease with factor VIII/von Willebrand factor concentrates: results from current studies and surveys. Blood Coagul Fibrinolysis 2005;16(Suppl 1):S17-S21
3. Federici AB. Management of inherited von Willebrand disease in 2006. Semin Thromb Hemost 2006;32:616-620
4. Pasi KJ, Collins PW, Keeling DM, et al. Management of von Willebrand disease: a guideline from the UK Haemophilia Centre Doctor's Organization. Haemophilia 2004;10:218-231
5. Mannucci PM. Scientific and Standardization Committee Communication. Recommended protocol for the study of the ex vivo biological effects of virus-inactivated plasma concentrates in patients with von Willebrand disease. Thromb Haemost 1992;68:84-87
6. Committee for Proprietary Medicinal Products. Guideline on the clinical investigation of human plasma derived von Willebrand factor products (CPMP/BPWG/220/02). 2005. Available at: http://www.emea.europa.eu/pdfs/human/ bpwg/022002en.pdf. Accessed November 22, 2007
7. Goudemand J, Mazurier C, Marey A, et al. Clinical and biological evaluation in von Willebrand's disease of a von Willebrand factor concentrate with low factor VIII activity. Br J Haematol 1992;80:214-221
8. Menache D, Aronson D, Darr F, et al. Pharmacokinetics of von Willebrand factor and factor VIIIC in patients with severe von Willebrand disease (type 3 VWD): estimation of the rate of factor VIIIC synthesis. Br J Haematol 1996;94:740-745
9. Menache D. Pharmacokinetics of von Willebrand factor and FVIII coagulant activity in patients with von Willebrand disease type 2 and 3. Haemophilia 1998;4(Suppl 3):44-47
10. Dobrkovska A, Krzensk U, Chediak JR. Pharmacokinetics, efficacy and safety of Humate-P in von Willebrand disease. Haemophilia 1998;4(Suppl 3):33-39
11. Lubetsky A, Martinowitz U, Luboshitz J, Kenet G, Keller N, Tamarin I. Efficacy and safety of a factor VIII-von Willebrand factor concentrate 8Y: stability, bacteriological safety, pharmacokinetic analysis and clinical experience. Haemophilia 2002;8:622-628
12. Auerswald G, Eberspacher B, Engl W, et al. Successful treatment of patients with von Willebrand disease using a high-purity double-virus inactivated factor concentrate (Immunate®). Semin Thromb Hemost 2002;28:203-213
13. Mannucci PM, Chediak J, Hanna W, et al; Alphanate Study Group. Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate: a prospective, multicenter study. Blood 2002;99:450-456
14. Ver Eist KMM, van Vliet HDM, Kappers-Klunne MC, Leebeek FWG. In vitro studies, pharmacokinetic studies and clinical use of a high purity double virus inactivated FVIII/VWF concentrate (Immunate) in the treatment of von Willebrand disease. Thromb Haemost 2004;92:67-74
15. Goudemand J, Scharrer I, Berntorp E, et al. Pharmacokinetic studies on Wilfactin, a von Willebrand factor concentrate with a low factor VIII content treated with three virus-inactivation/removal methods. J Thromb Haemost 2005;3:2219-2227
16. Lethagen S, Kyrle PA, Castaman G, Haertel S, Mannucci PM. for the HAEMATE® P Surgical Study Group, von Willebrand factor/factor VIII concentrate (Haemate® P) dosing based on pharmacokinetics: a prospective multicenter trial in elective surgery. J Thromb Haemost 2007;5:1420-1430
17. Favaloro EJ, Lloyd J, Rowell J, et al. Comparison of the pharmacokinetics of two von Willebrand factor concentrates (Biostate® and AHF (High Purity)) in people with von Willebrand disorder: A randomised cross-over, multi-centre study. Thromb Haemost 2007;97:922-930
18. Favaloro EJ, Bukuya M, Martinelli T, et al. A comparative multi-laboratory assessment of factor concentrate and implications for clinical efficacy as potential replacement therapy in von Willebrand's Disease (VWD). Thromb Haemost 2002;87:466-476
19. Oates A, Polmear E, Herrington R, et al. Willebrand factor characterization of a severe dry-heat treated factor VIII concentrate, AHF (high purity). Thromb Res 1992;65:389-399
20. Shortt J, Dunkley S, Rickard K, Baker R, Street A. Efficacy and safety of a high purity, double virus inactivated factor VIII/von Willebrand factor concentrate (Biostate®) in patients with von Willebrand disorder requiring invasive or surgical procedures. Haemophilia 2007;13:144-148
21. Favaloro EJ. von Willebrand factor (VWF) collagen binding (activity) assay (VWF:CBA) in the diagnosis of von Willebrand's disorder (VWD): A 15-year journey. Semin Thromb Hemost 2002;28:191-202
22. Favaloro EJ. Laboratory identification of von Willebrand disease: technical and scientific perspectives. Semin Thromb Hemost 2006;32:456-471
23. Federici AB, Canciani MT, Forza I, Cozzi G. Ristocetin cofactor and collagen binding activities normalized to antigen levels for a rapid diagnosis of type 2 von Willebrand disease. Single center comparison of four different assays. Thromb Haemost 2000;84:1127-1128
24. Baronciani L, Federici AB, Cozzi G, Canciani MT, Mannucci PM. von Willebrand factor collagen binding assay in von Willebrand disease type 2A, 2B, and 2M. J Thromb Haemost 2006;4:2088-2090
25. Hubbard AR. von Willebrand factor standards for plasma and concentrate testing. Semin Thromb Hemost 2006;32:522-528
26. Budde U, Metzner HJ, Muller H-G. Comparative analysis and classification of von Willebrand factor/factor VIII concentrates: impact on treatment of patients with von Willebrand disease. Semin Thromb Hemost 2006;32:626-635
27. Mazurier C. Composition, quality control, and labeling of plasma-derived products for the treatment of von Willebrand disease. Semin Thromb Hemost 2006;32:529-536
28. Favaloro EJ, Facey D, Grispo L. Laboratory assessment of von Willebrand Factor: use of different assays can influence the diagnosis of von Willebrand's disease, depending on differing sensitivity to sample preparation and differential recognition of high molecular weight VWF forms. Am J Clin Pathol 1995;104:264-271
29. Favaloro EJ. Collagen binding assay for von Willebrand factor (VWF:CBA): detection of von Willebrand's disease (VWD), and discrimination of VWD subtypes, depends on collagen source. Thromb Haemost 2000;83:127-135
30. Neugebauer BM, Goy C, Budek I, Seitz R. Comparison of two von Willebrand factor collagen binding assays with different affinities for low, medium and high multimers of von Willebrand factor. Semin Thromb Hemost 2002;28:139-147
31. Neugebauer BM, Goy C, Seitz R. A collagen binding assay: an additional method for von Willebrand factor activity in therapeutic concentrates. Thromb Haemost 2002;88:871-872
32. st International Standard for von Willebrand Factor Concentrate (00/514). Thromb Haemost 2002;88:380-386
33. Favaloro EJ. An update on the von Willebrand factor collagen binding assay (VWF:CB) - 21 years of age and beyond adolescence, but not yet a mature adult. Semin Thromb Hemost 2007;33:727-744
34. Favaloro EJ. Standardization, regulation, quality assurance and emerging technologies in hemostasis: Issues, controversies, benefits and limitations. Semin Thromb Hemost 2007;33:290-297
35. Hubbard A. Minutes of the International Society on Thrombosis and Haemostasis (ISTH) von Willebrand factor (VWF) Scientific Standardisation Committee (SSC) meeting. Available at: http://www.med.unc.edu/isth/ssc/ 07sscminutes/07vwf.htm. Accessed September 4, 2007