Blood Products for Hemophilia

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Montreal: World Federation of Hemophilia, 2003. Also available as a PDF file at www.wfh.org DeBiasi R, Rocino A, Miraglia E, et al. The impact of a very high purity factor VIII concentrate on the immune system of human immunodeficiency virus-infected hemophiliacs: a randomized, prospective, two-year comparison with an intermediate purity concentrate. Blood 1991; 78: 1919–22 Goedert JJ, Cohen AR, Kessler CM. Risks of immunodeficiency, AIDS and death related to purity of factor VIII concentrate: Multicenter Hemophilia Cohort Study. Lancet 1994; 344: 791–2 Sabin C, Pasi J, Phillips A, et al. CD4+ counts before and after switching to monoclonally high-purity factor VIII concentrate in HIV-infected haemophilic patients. Thromb Haemost 1994; 72: 214–7 Hay CR, Ludlam CA, Lowe GD, et al. The effect of monoclonal or ion-exchange purified factor VIII concentrate on HIV disease progression: a prospective cohort comparison. Br J Haematol 1998; 101: 632–7 Thomas DP, Hampton KK, Dasani H, et al. A cross-over pharmacokinetic and thrombogenicity study of a prothrombin complex concentrate and a purified factor IX concentrate. Br J Haematol 1994; 87: 782–8 Kasper CK. Thromboembolic complications. Thromb Diath Haemorrh 1975; 33: 640–4 Conlan MG, Hoots WK. Disseminated intravascular coagulation and hemorrhage in hemophilia B following elective surgery. Am J Hematol 1990; 35: 203–7 Fuerth JH, Mahrer P. Myocardial infarction after factor IX therapy. JAMA 1981; 245: 1455–6 Horowitz MS, Rooks C, Horowitz B, et al. Virus safety of solvent/detergent-treated antihaemophilic factor concentrate. Lancet 1988; II: 186–9 Schimpf K, Brackmann HH, Kreuz W, et al. Absence of anti-human immunodeficiency virus types 1 and 2 seroconversion after the treatment of hemophilia A or von Willebrand’s disease with pasteurized factor VIII concentrate. N Engl J Med 1989; 321: 1148–52 Rizza CR, Fletcher ML, Kernoff PB. Confirmation of viral safety of dry heated factor VIII concentrate (8Y) prepared by Bio Products Laboratory (BPL): a report on behalf of UK Haemophilia Centre Directors. Br J Haematol 1993; 84: 269–72 Vermylen J, Peerlinck K. Review of the hepatitis A epidemics in hemophiliacs in Europe. Vox Sang 1994; 67Suppl. 4: 8–11 Soucie JM, Roberston BH, Bell BP, et al. Hepatitis A virus infections associated with clotting factor concentrate in the United States. Transfusion 1998; 38: 573–9 Yee TT, Cohen BJ, Pasi KJ, et al. Transmission of symptomatic parvovirus parvovirus B19 infection by clotting factor concentrate. Br J Haematol 1996; 93: 457–9 Azzi A, Morfini M, Mannucci PM. The transfusion-associated transmission of parvovirus B19. Transfus Med Rev 1999; 13: 194–204 Farrugia A. Risk of variant Creutzfeldt-Jakob disease from factor concentrates: current perspectives. Haemophilia 2002; 8: 230–5 Burnouf T, Radosevich M. Nanofiltration of plasma-derived biopharmaceutical products. Haemophilia 2003; 9: 24–37 United Kingdom Haemophilia Centre Doctors’ Organisation (UKHCDO). Guidelines on the selection and use of therapeutic products to treat haemophilia and other hereditary bleeding disorders. Haemophilia 2003; 9: 1–23 Makris M, Conlon CP, Watson HG. Immunization of patients with bleeding disorders. Haemophilia 2003; 9: 541–6 Jantsch-Plunger V, Beck G, Maurer W. PCR detection of a low viral load in a prothrombin complex concentrate that transmitted hepatitis B virus. Vox Sang 1995; 69: 352–4 Giangrande PLF. Treatment of hemophilia: recombinant products only? Yes. J Thromb Haemost 2003; 1: 214–5 Mannucci PM. Treatment of hemophilia: recombinant products only? No. J Thromb Haemost 2003; 1: 216–7 Eis-Hübinger AM, Sasowski U, Brackmann HH, et al. Parvovirus B19 DNA is frequently present in recombinant coagulation factor VIII products [letter]. Thromb Haemost 1996; 76: 1120 Azzi A, DeSantis R, Morfini M, et al. TT virus contaminates first-generation recombinant factor VIII concentrates. Blood 2001; 15: 2571–3 Makris M, Garson JA, Ring CJ, et al. Hepatitis C viral RNA in clotting factor concentrates and the development of hepatitis in recipients. Blood 1993; 81: 1898–902 Hunter N, Foster J, Chong A, et al. Transmission of prion diseases by blood transfusion. J Gen Virol 2002; 83: 2897–905 Giangrande PLF, Taylor S. Communicating risk: the example of vCJD. Proceedings of the World Federation of Haemophilia Global Forum on the Safety and Supply of Hemophilia Treatment Products. Montreal: World Federation of Haemophilia, 2002: 39 Phillips Report on the BSE Inquiry in the UK (2000) [online]. Available from URL: www.bseinquiry.gov.uk/report/volume10/chaptef2.htm#260106 [Accessed 2004 May 27] Lusher JM. Factor VIII inhibitors with recombinant products: prospective clinical trials. Haematologica 2000; 85Suppl. 10: 2–6 Mauser-Bunschoten EP, van der Born JG, Bongers M, et al. Purity of factor VIII product and incidence of inhibitors in previously untreated patients with haemophilia A. Haemophilia 2001; 7: 364–8 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 Behrmann M, Pasi J, Saint-Remy J-M, et al. Von Willebrand factor modulates factor VIIII immunogenicity: comparative study of different factor VIII concentrates in a haemophilia A mouse model. Thromb Haemost 2002; 88: 221–229 Auerswald G, Spranger T, Brackmann H-H. The role of plasma-derived factor VIII/von Willebrand factor concentrates in the treatment of hemophilia A patients. Haematologica 2003; 88Suppl. 9: 21–5 Giles AR, Rivard GE, Teitel J, et al. Surveillance for factor VIII inhibitor development in the Canadian hemophilia A population following the wide-spread introduction of recombinant factor VIII replacement therapy. Transfus Sci 1998; 19: 139–48 Poon M-C, Lillicrap D, Hensman C, et al. Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study. Thromb Haemost 2002; 87: 431–5 Oldenburg J, El-Maari O, Schwaab R. Inhibitor development in correlation to factor VIII genotypes. Haemophilia 2002; 8Suppl. 2: 23–9 Barrowcliffe TW. Standardization of FVIII and FIX assays. Haemophilia 2003; 9: 397–402 Hubbard AR, Weller LJ, Bevan SA. A survey of one-stage and chromogenic potencies in therapeutic factor VIII concentrates. Br J Haematol 2002; 117: 247–8 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 Barrowcliffe TW, Raut S, Hubbard AR. Discrepancies in potency assessment of recombinant FVIII concentrates. Haemophilia 1998; 4: 634–40 Mikaelsson M, Oswaldsson U, Sandberg H. Influence of phospholipids on the assessment of factor VIII activity. Haemophilia 1998; 4: 646–50 Mikaelsson M, Oswaldsson U, Jankowski MA. Measurement of factor VIII activity of B-domain deleted recombinant factor VIII. Semin Hematol 2001; 38Suppl. 4: 13–23 Szende A, Schramm W, Flood E, et al. Health-related quality of life assessment in adult haemophilia patients: a systematic review and evaluation of instruments. Haemophilia 2003; 9: 678–87 Bullinger M, Von Mackensen S, Fischer K, et al. Pilot testing of the ‘Haemo-QoL’ quality of life questionnaire for haemophiliac children in six European countries. Haemophilia. 2002; 8Suppl. 2: 47–54 Gringeri A, Mantovani LG, Scalone L, et al. Cost of care and quality of life in hemophilia complicated by inhibitors: the COCIS Study Group. Blood 2003; 102: 2358–63 O’Mahony B. Global haemophilia care: future challenges and opportunities [abstract]. Haemophilia 2002; 8: 515 World Health Organization (WHO). Delivery of treatment for haemophilia: report of a joint WHO/WFH/ISTH meeting; 2002 Feb 11–12; London Rock G, Berger R, Lange J, et al. A novel, automated method of temperature cycling to produce cryoprecipitate. Transfusion 2001; 41: 232–5 Evatt BL, Austin H, Leon G, et al. Haemophilia therapy: assessing the cumulative risk of HIV exposure by cryoprecipitate. Haemophilia 1999; 5: 295–300 Saenko EL, Ananyeva NM, Moayeri M, et al. Development of improved factor VIII molecules and new gene transfer approaches for hemophilia A. Curr Gene Ther 2003; 3: 27–41 Saenko EL, Ananayeva NM, Shima M, et al. The future of recombinant coagulation factor proteins. J Thromb Haemost 2003; 1: 922–30 Pipe SW, Kaufman RJ. Characterization of a genetically engineered inactivation-resistant coagulation factor VIIIa. Proc Natl Acad Sci U S A 1997; 94(22): 11851–6 Sarafanov AG, Ananyeva NM, Shima M, et al. Cell surface heparin sulphate proteoglycans participate in factor VIII catabolism mediated by low density lipoprotein receptor-related protein. J Biol Chem 2001; 276: 11970–9 Barrow RT, Healey JF, Gailani D, et al. Reduction of the antigenicity of factor VIII toward complex inhibitory antibody plasmas using multiply-substituted hybrid human/porcine factor VIII molecules. Blood 2000; 95: 564–8 Miao HZ, Sirachainan N, Palmer L, et al. Bioengineering of coagulation factor VIII for improved secretion. Blood 2004 May 1; 103(9): 3412–9 Paleyanda RK, Velander WH, Lee TK, et al. Transgenic pigs produce functional human factor VIII in milk. Nat Biotechnol 1997; 15: 971–5 Pollock D, Kutzko JP, Birck-Wilson E, et al. Transgenic milk as a method for the production of recombinant antibodies. J Immunol Methods 1999; 231: 147–57 Konkle BA, Bauer KA, Weinstein R, et al. Use of recombinant human antithrombin in patients with congenital antithrombin deficiency undergoing surgical procedures. Transfusion 2003; 43: 390–4 Colman A. Dolly, Polly and other ‘ollys’: likely impact of cloning technology on biomedical uses of livestock. Genet Anal 1999; 15: 167–73 Tebbutt SJ. Technology evaluation: transgenic alpha-1-antitrypsin (AAT), PPL therapeutics. Curr Opin Mol Ther 2000; 2: 199–204 Lee JW, Wu SC, Tian XC, et al. Production of cloned pigs by whole-cell intracytoplasmic microinjection. Biol Reprod 2003; 69: 995–1001 Vandendriessche T, Collen D, Chuah MKL. Gene therapy for the hemophilias. J Thromb Haemost 2003; 1: 1550–15558 Kay MA, Manno CS, Ragni MV, et al. Evidence for gene transfer and expression of factor IX in haemophilia B treated with an AAV vector. Nat Genet 2000; 24: 257–61 High KA. Adeno-associated virus-mediated gene transfer for hemophilia B. Int J Hematol 2002; 76: 310–8 Roth DA, Tawa NE, O’Brien J, et al. Non viral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med 2001; 344: 1735–42