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Thromb Haemost 1995; 74(04): 1191-1196
DOI: 10.1055/s-0038-1649902
Scientific And Standardization Committee Communications
Schattauer GmbH Stuttgart

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

On behalf of the Subcommittee on Factor VIII and IX of the Scientific and Standardization Committee of the ISTH
C V Prowse
The Scottish National Blood Transfusion Service, National Science Laboratory, Edinburgh, UK
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Publication Date:
09 July 2018 (online)

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Summary

In the therapy of factor VIII deficiency, experience has taught the value of achieving a target level of plasma coagulation factor VIII. There is a consensus that an empirical value of 2 iu/dl/iu/kg may be used to estimate recovery, and hence plasma levels, of factor VIII. This may be influenced by the methods used to assign label potency to the products and for assessment of patient post-infusion plasmas. To assess a possible influence of the standard used to assign product potency on in vivo recovery, a survey was undertaken of recent in vivo recovery studies. Analysis of submitted data, in combination with published studies over the last ten years, revealed a significant influence of the standard used to assign potency to products on the measured in vivo recovery. Furthermore, from limited data the potency determined in post-infusion patient plasmas was found not to be influenced by the use of one-stage or chromogenic assay methods.

 

  • References

  • 1 Abilgaard CF, Simone JV, Corrigan JJ, Seeler RA, Edelstein G, Vanderhei-den BA, Schulman I. Treatment of haemophilia with glycine-precipitated factor VIII. New Eng J Med 1966; 275: 471-475
    CrossrefPubMedSearch in Google Scholar
  • 2 Shanbrom E, Thelin G. Experimental prophylaxis of severe haemophilia A with a factor VIII concentrate. JAMA 1969; 208: 1853-1856
    CrossrefPubMedSearch in Google Scholar
  • 3 Committee for Proprietary Medicinal Products (1992). . Coagulation Factor VIII Concentrate: Summary of Product Characteristics. Publ: European Union III/3361/92EN.
    PubMed
  • 4 Lee ML, Gomperts ED, Kingdon HS. A note on the calculation of recovery for factor VIII. Thromb Haemost 1993; 69: 87
    Thieme ConnectPubMedSearch in Google Scholar
  • 5 Lusher JM, Ofosu FA, Edson JR, Joist JH, Chavin SI, Weiss AE, Hillman CR. North American study of factor VIII concentrate potency. Scand J Haematol 1984; 33 suppl 40: 149-160
    PubMedSearch in Google Scholar
  • 6 Lusher JM. Factor VIII concentrates: Matching what you see with what you get. JAMA 1985; 254: 802-803
    CrossrefPubMedSearch in Google Scholar
  • 7 Kasper CK. In vivo recovery of FVIII:C in haemophilia patients. Scand J Haematol 1984; 33 suppl 41: 147-154
    PubMedSearch in Google Scholar
  • 8 Schimpf K, Rothmann P, Schumacher K. Comparison of factor VIII concentrates in non-bleeding patients. Scand J Haematol 1984; 33 suppl 41: 131-145
    PubMedSearch in Google Scholar
  • 9 Morfini M, Longo G, Matucci M, Vannini S, Messori A, Filimber E, Duminuco M, Avanzi G, Rossifer P. Cryoprecipitate and factor VIII commercial concentrates: in vitro characteristics and in vivo compartmental analysis. La Ricerca Clin Lab 1984; 14: 681-691
    PubMedSearch in Google Scholar
  • 10 Kjellman H. Calculations of factor VIII in vivo recovery and half-life. Scand J Haematol 1984; 33 suppl 40: 165-174
    PubMedSearch in Google Scholar
  • 11 Ofosu FA, Blajchman MR, Barr R, Inwood M. Approaches to the measurement of the in vivo response to factor VIII concentrate infusion. Thromb Res 1985; 39: 429-437
    CrossrefPubMedSearch in Google Scholar
  • 12 Matucci M, Messori A, Donatico G, Longo G, Vannini S, Morfini M, Tendi E, Rossifer PL. Kinetic evaluation of four factor VIII concentrates by model independent methods. Scand J Haematol 1985; 34: 22-28
    PubMedSearch in Google Scholar
  • 13 Messori A, Longo G, Matucci M, Morfini M, Ferrini PLR. Clinical pharmacokinetics of factor VIII in patients with classic haemophilia. Clin Pharmacokinetic 1987; 13: 365-380
    CrossrefPubMedSearch in Google Scholar
  • 14 Longo G, Matucci M, Messori A, Morfini M, Rossifer P. Pharmacokinetics of a new heat treated concentrate of factor VIII estimated by model independent methods. Thromb Res 1986; 42: 471-476
    CrossrefPubMedSearch in Google Scholar
  • 15 Blomback M, Kjellman H, Allain JP, Hedner U, Schimpf K, Wiechel B. On the unreliability of one-stage factor VIII:C clotting assays after infusion of factor VIII:C concentrates. Scand J Clin Lab Inv 1987; 47: 561-566
    CrossrefPubMedSearch in Google Scholar
  • 16 Brettler DB, Forsberg AD, Levine PH, Petillo J, Lamon K, Sullivan JL. Factor VIII :C concentrate purified from plasma using monoclonal antibodies: human studies. Blood 1989; 73: 1859-1865
    PubMedSearch in Google Scholar
  • 17 Mazurier C, Parquet-Gemez A, Goudemand M. Validation of a procedure for potency assessing of a high purity factor VIII concentrate - comparison of different factor VIII coagulant assays and effect of prediluent. Thromb Haemost 1990; 64: 251-255
    Thieme ConnectPubMedSearch in Google Scholar
  • 18 Nilsson IM, Berntorp E. Clinical efficacy of clotting factor concentrates: survival, recovery and hemostatic efficacy. In: Coagulation and Blood Transfusion. Smit-Sibinga CTh, Das PC, Mannucci PM. Publ: Kluwer Academic; eds pp 193-206
  • 19 Schwartz RS, Abildgaard CF, Aledort LM, Arkin S, Bloom AL, Brack-mann HH, Brettler DB, Fukui H, Hilgartner MW, In wood MJ, Kasper CK, Kernoff PB A, Levine PH, Lusher JM, Mannucci PM, Scharrer J, MacKenzie MA, Pancham N, Kuo HS, Allred RU. Human recombinant DNA derived antihemophilic factor in the treatment of hemophilia A. New Engl J Med 1990; 323: 1800-1805
    CrossrefPubMedSearch in Google Scholar
  • 20 Morfini M, Mannucci PM, Longo G, Cinotti S, Messori A. Comparative evaluation of the pharmacokinetics of three monoclonal factor VIII concentrates. Thromb Res 1991; 61: 285-290
    CrossrefPubMedSearch in Google Scholar
  • 21 Peerlinck K, Arnout J, Tamise M, Vanherle A, Fondu P, Vermylen J. In vitro and in vivo evaluation of two factor VIII concentrates virally inactivated by solvent detergent or pasteurisation. Acta Clin Belg 1991; 46: 298-304
    CrossrefPubMedSearch in Google Scholar
  • 22 Kasper C, Kim HC, Gomperts ED, Smith KJ, Salzman PM, Tipping D, Miller R, Montgomery RM. In vivo recovery and survival of monoclonal antibody purified factor VIII:C concentrates. Thromb Haemost 1991; 66: 730-733
    Thieme ConnectPubMedSearch in Google Scholar
  • 23 Morfini M, Longo G, Messori A, Lee M, White G. Mannucci P and the Recombinant Study Group. Pharmacokinetic properties of recombinant factor VIII compared with a monoclonally purified concentrate (Hemofil M) Thromb Haemost 1992; 68: 433-435
    Thieme ConnectPubMedSearch in Google Scholar
  • 24 Addiego JE, Gomperts E, Lui S-L, Bailey P, Courter SG, Lee ML, Neslund GG, Kingdon HS, Griffith MJ. Treatment of hemophilia A with a highly purified factor VIII concentrate prepared by anti-VIII:C immunoaffinity chromatography. Thromb Haemost 1992; 67: 19-27
    Thieme ConnectPubMedSearch in Google Scholar
  • 25 Messori A, Morfini M, Blombäck M, Cinotti S, Longo G, Schimpf K, Schumacher K, Novakova-Banet A, Delvos U, Kjellman H. Pharmacokinetics of two pasteurised factor VIII concentrates by different and multicentre assays of factor VIII activity. Thromb Res 1992; 65: 699-708
    CrossrefPubMedSearch in Google Scholar
  • 26 Smith KJ, Lusher JM, Cohen AR, Salzman P. Initial clinical experience with a new pasteurised monoclonal antibody purified factor VIII: C. Sem Haematol 1990; 27 suppl 2: 25-29
    PubMedSearch in Google Scholar
  • 27 Perret BA, Poorbeik M, Morell A. Klinische Prüfung von Premofil M SRK, einem mit monoklonalen Antikörpern hochgereinigten Gerinnungsfaktor- VIII-Konzentrat aus Humanplasma. Schweiz Med Wochenschrift 1991; 121: 1624-1627
    PubMedSearch in Google Scholar
  • 28 Rousell R, Kasper C, Schwarz R. The pharmacology of a new pasteurised antihemophilic factor concentrate derived from human blood plasma. Transfusion 1989; 29: 208-221
    CrossrefPubMedSearch in Google Scholar
  • 29 Berntorp E, Nilsson I. Biochemical and in vivo properties of commercial virus inactivated factor VIII concentrates. Eur J Haematol 1988; 40: 205-214
    PubMedSearch in Google Scholar
  • 30 Nilsson I. Biochemical properties of Monoclate factor VIII: C antihaemophilic factor (human) using monoclonal antibody to von Willebrand factor (vWf). Sem Haem 1988; 25 suppl 1: 33-37
    PubMedSearch in Google Scholar
  • 31 Hellstern P, Kiehl R, Miyashita C, Schwendt H, von Blohn G, Kohler M, Buttner M, Wenzel E. Factor VIII:C recovery and half life after infusion of steam-treated high purity factor VIII concentrate in severe haemophilia A: comparison of one-stage assay, two-stage assay and a chromogenic substrate assay. Thromb Haemost 1986; 56: 353-359
    Thieme ConnectPubMedSearch in Google Scholar
  • 32 Rock G, Tittley P, Fuller V. An in vivo assessment of factor VIII concentrates. JAMA 1985; 254: 777-780
    CrossrefPubMedSearch in Google Scholar
  • 33 Knevelman A, de Wit HJ C, Potstra P, Does JA vd. Development and small scale production of a severely heated factor VIII concentrate. Vox Sang 1994; 66: 89-93
    CrossrefPubMedSearch in Google Scholar
  • 34 Hiemstra H, Nieuweboer CE F, Idoe MA, Claassen JE, Vos AH V, Tersmette M, Strengers PF W, Over J, Mauser-Bunschoten EP, Heijboer H, Holdrinet AC J M. Evaluation of wet pasteurisation of a Factor VIII concentrate produced by controlled pure silica adsorption. Folia Haematol 1990; 117: 557-563
    PubMedSearch in Google Scholar
  • 35 Kogenate. 1993 Product Insert.
    PubMedSearch in Google Scholar
  • 36 Recombinate. 1992 Product Insert.
    PubMedSearch in Google Scholar
  • 37 Walker IR, Pai MK R, Blair BA, Johnston MA, Birkett NJ, Ofosu FA, Naylor DH. Factor VIII concentrate for haemophilia: comparison of two- heated products. Can Med AJ 1987; 136: 150-152
    PubMedSearch in Google Scholar
  • 38 Berntorp E, Brackmann HH, Dobrovska A, Lethagen S, Morfini M, Oldenburg J. Comparative evaluation of the pharmacokinetic characteristics of two pasteurised factor VIII concentrates. World Federation of Haemophilia; Mexico: 04/1994. abstract 213
    Search in Google Scholar
  • 39 Shurafa M. Discrepant in vivo recovery of factor VIII: C following infusion of two different monoclonal factor VIII preparations. World Federation of Haemophilia; Mexico: 04/1994. abstract 220
    Search in Google Scholar
  • 40 Bray GL, Lee ML, Buckwalter C, Lynes M, Courter S, Gomperts ED. Use of recombinant factor VIII Recombinate in previously untreated patients with haemophilia A. World Federation of Haemophilia; Mexico: 04/1994. abstract 107
    Search in Google Scholar
  • 41 Allain JP, Verroust F, Soulier JP. One or two stage assay for factor VIII. Lancet 1979; i: 1076-1077
    PubMedSearch in Google Scholar
  • 42 Nilsson IM, Kirkwood TB L, Barrowcliffe TW. In vivo recovery of factor VIII: a comparison of one-stage and two-stage assay methods. Thromb Haemost 1979; 42: 1230-1239
    Thieme ConnectPubMedSearch in Google Scholar
  • 43 Mikaelsson M, Oswaldsson U, Nilsson IM. Potency and in vivo recovery of high purity factor VIII concentrate. Thromb Haemost 1994; 72: 160-161
    Thieme ConnectPubMedSearch in Google Scholar