BAY 81-8973, a full-length recombinant factor VIII: Results from an International comparative laboratory field study

Haemophilia

Volumn 22, Issue 3, 2016, Pages e192-e199

  Kitchen, S ^a   Beckmann, H ^b   Katterle, Y ^b   Bruns, S ^c   Tseneklidou Stoeter, D ^b   Maas Enriquez, M ^b  

^a ROYAL HALLAMSHIRE HOSPITAL   (United Kingdom)

^b BAYER PHARMA AG   (Germany)

^c Winicker Norimed GmbH   (Germany)

Author keywords

Chromogenic assay; Factor VIII; Field study; One stage assay; Postinfusion monitoring; Recombinant factor VIII

Indexed keywords

RECOMBINANT BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8; DRUG; F8 PROTEIN, HUMAN; RECOMBINANT PROTEIN;

ANALYTICAL PARAMETERS; ARTICLE; BLOOD CLOTTING TEST; CALIBRATION; CHROMOGENIC ASSAY; CONCENTRATION (PARAMETERS); CONTROLLED STUDY; DIAGNOSTIC KIT; FIELD STUDY; INTERLABORATORY VARIABILITY; INTRALABORATORY VARIABILITY; LABORATORY; MEASUREMENT ACCURACY; ONE STAGE ASSAY; PLASMA; PRIORITY JOURNAL; CHEMISTRY; COMPARATIVE STUDY; HEMOPHILIA A; HUMAN; INTERNATIONAL COOPERATION; OBSERVER VARIATION;

FACTOR VIII; HEMOPHILIA A; HUMANS; INTERNATIONAL COOPERATION; LABORATORIES; OBSERVER VARIATION; PHARMACEUTICAL PREPARATIONS; PLASMA; RECOMBINANT PROTEINS;

EID: 84962877636     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.12925     Document Type: Article

References (28)

1. Srivastava A, Brewer AK, Mauser-Bunschoten EP et al. Guidelines for the management of hemophilia. Haemophilia 2013; 19: e1-47.
2. Dodt J, Hubbard AR, Wicks SJ et al. Potency determination of factor VIII and factor IX for new product labelling and postinfusion testing: challenges for caregivers and regulators. Haemophilia 2015; 21: 543-9.
3. Kitchen S, Signer-Romero K, Key NS. Current laboratory practices in the diagnosis and management of haemophilia: a global assessment. Haemophilia 2015; 21: 550-7.
4. Makris M, Peyvandi F. Assaying FVIII activity: one method is not enough, and never was. Haemophilia 2014; 20: 301-3.
5. Mikaelsson M, Oswaldsson U, Sandberg H. Influence of phospholipids on the assessment of factor VIII activity. Haemophilia 1998; 4: 646-50.
6. Gu JM, Ramsey P, Evans V et al. Evaluation of the activated partial thromboplastin time assay for clinical monitoring of PEGylated recombinant factor VIII (BAY 94-9027) for haemophilia A. Haemophilia 2014; 20: 593-600.
7. Barrowcliffe TW, Raut S, Sands D, Hubbard AR. Coagulation and chromogenic assays of factor VIII activity: general aspects, standardization, and recommendations. Semin Thromb Hemost 2002; 28: 247-56.
8. Mikaelsson M, Oswaldsson U, Jankowski MA. Measurement of factor VIII activity of B-domain deleted recombinant factor VIII. Semin Hematol 2001; 38: 13-23.
9. Hubbard AR. Potency labeling of novel factor VIII and factor IX concentrates: past experience and current strategy. Semin Thromb Hemost 2015; 41: 849-54.
10. Assay of human coagulation factor VIII (2.7.4). European Pharmacopoeia, 5th edn. Strasbourg, France: Council of Europe, 2005: 194-5.
11. National Hemophilia Foundation. Medical and Scientific Advisory Council (MASAC) statement regarding use of various clotting factor assays to monitor factor replacment therapy. Document #228. Available at http://www.hemophilia.org/sites/default/files/document/files/masac-228.pdf. Accessed October 5, 2015.
12. Lusher JM, Hillman-Wiseman C, Hurst D. In vivo recovery with products of very high purity-assay discrepancies. Haemophilia 1998; 4: 641-5.
13. Barrowcliffe TW, Hubbard AR, Kitchen S. Standards and monitoring treatment. Haemophilia 2012; 18: 61-5.
14. Jennings I, Kitchen DP, Woods TA, Kitchen S, Walker ID. Emerging technologies and quality assurance: the United Kingdom National External Quality Assessment Scheme perspective. Semin Thromb Hemost 2007; 33: 243-9.
15. Ingerslev J, Jankowski MA, Weston SB, Charles LA. ReFacto Field Study Participants. Collaborative field study on the utility of a BDD factor VIII concentrate standard in the estimation of BDDr factor VIII: C activity in hemophilic plasma using one-stage clotting assays. J Thromb Haemost 2004; 2: 623-8.
16. Pouplard C, Caron C, Aillaud MF et al. The use of the new ReFacto AF Laboratory Standard allows reliable measurement of FVIII: C levels in ReFacto AF mock plasma samples by a one-stage clotting assay. Haemophilia 2011; 17: e958-62.
17. 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.
18. Lee CA, Owens D, Bray G et al. Pharmacokinetics of recombinant factor VIII (recombinate) using one-stage clotting and chromogenic factor VIII assay. Thromb Haemost 1999; 82: 1644-7.
19. Morfini M, Cinotti S, Bellatreccia A et al. A multicenter pharmacokinetic study of the B-domain deleted recombinant factor VIII concentrate using different assays and standards. J Thromb Haemost 2003; 1: 2283-9.
20. Cauchie M, Toelen J, Peerlinck K, Jacquemin M. Practical and cost-effective measurement of B-domain deleted and full-length recombinant FVIII in the routine haemostasis laboratory. Haemophilia 2013; 19: e133-8.
21. Kitchen S, Gray E, Mertens K. Monitoring of modified factor VIII and IX products. Haemophilia 2014; 20: 36-42.
22. Vogel JH, Huesslein A, Goudar C et al. BAY 81-8973 - a new full-length recombinant FVIII product using novel manufacturing technologies. Haemophilia 2010; 16: 40.
23. Saxena K, Lalezari S, Oldenburg J et al. Pharmacokinetics, efficacy, and safety of BAY 81-8973, a full-length plasma-protein-free recombinant factor VIII product: results from the LEOPOLD I trial. J Thromb Haemost 2013; 11: 928.
24. Kavakli K, Yang R, Rusen L, Beckmann H, Tseneklidou-Stoeter D, Maas Enriquez M. Prophylaxis versus on-demand treatment with BAY 81-8973, a full-length plasma-protein-free rFVIII product: results from a randomized trial (LEOPOLD II). J Thromb Haemost 2015; 13: 360-9.
25. Ljung R, Kenet G, Mancuso ME et al. BAY 81-8973 safety and efficacy for prophylaxis and treatment of bleeds in previously treated children with severe hemophilia A: results of the LEOPOLD Kids trial. Haemophilia 2015. doi: 10.1111/hae.12866.
26. Oldenburg J, Lalezari S, Rusen L et al. Efficacy and safety of BAY 81-8973, Bayer's new full-length recombinant factor VIII: results from the LEOPOLD I and II trials. Presented at: German, Austrian, and Swiss Society of Thrombosis and Haemostasis Research; February 24-27, 2015; Düsseldorf, Germany.
27. Viuff D, Barrowcliffe T, Saugstrup T, Ezban M, Lillicrap D. International comparative field study of N8 evaluating factor VIII assay performance. Haemophilia 2011; 17: 695-702.
28. Sommer JM, Moore N, McGuffie-Valentine B et al. Comparative field study evaluating the activity of recombinant factor VIII Fc fusion protein in plasma samples at clinical haemostasis laboratories. Haemophilia 2014; 20: 294-300.