Download PDF
Thromb Haemost 2015; 113(05): 911-914
DOI: 10.1160/TH15-04-0273
Invited Editorial Focus
Schattauer GmbH

Factor VIII products in haemophilia A: one size fits all?

Pier Mannuccio Mannucci
1   Scientific Direction, IRCCS Ca’ Granda Maggiore Policlinico Hospital, Milan, Italy
,
Isabella Garagiola
2   Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca’ Granda Maggiore Policlinico Hospital, Milan, Italy
› Author Affiliations
Further Information

Publication History

Received: 01 April 2015

Accepted 01 April 2015

Publication Date:
24 November 2017 (online)

    Permissions and Reprints
 

  • References

  • 1 Mannucci PM, Tuddenham EG. The haemophilias--from royal genes to gene therapy. N Engl J Med 2001; 344: 1773-1779.
    CrossrefPubMedGoogle Scholar
  • 2 Gouw SC, van der Bom JG, van den Berg HM. Treatment-related risk factors of inhibitor development in previously untreated patients with haemophilia A: the CANAL cohort study. Blood 2007; 109: 4648-4654.
    CrossrefPubMedGoogle Scholar
  • 3 Gouw SC, van den Berg HM, Fischer K. et al. Intensity of factor VIII treatment and inhibitor development in children with severe haemophilia A: the RODIN study. Blood 2013; 121: 4046-4055.
    CrossrefPubMedGoogle Scholar
  • 4 Xi M, Makris M, Marcucci M. et al. Inhibitor development in previously treated haemophilia A patients: a systematic review, meta-analysis, and meta-regression. J Thromb Haemost 2013; 11: 1655-1662.
    CrossrefPubMedGoogle Scholar
  • 5 Santagostino E, Mancuso ME, Rocino A. et al. Environmental risk factors for inhibitor development in children with haemophilia A: a case-control study. Br J Haematol 2005; 130: 422-427.
    CrossrefPubMedGoogle Scholar
  • 6 Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003; 09: 418-435.
    CrossrefPubMedGoogle Scholar
  • 7 Goudemand J, Laurian Y, Calvez T. Risk of inhibitors in haemophilia and the type of factor replacement. Curr Opin Hematol 2006; 13: 316-322.
    CrossrefPubMedGoogle Scholar
  • 8 Kaufman RJ, Wasley LC, Dorner AJ. Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells. J Biol Chem 1988; 263: 6352-6362.
    PubMedGoogle Scholar
  • 9 Hironaka T, Furukawa K, Esmon PC. et al. Comparative study of the sugar chains of factor VIII purified from human plasma and from the culture media of recombinant baby hamster kidney cells. J Biol Chem 1992; 267: 8012-8020.
    PubMedGoogle Scholar
  • 10 Grancha S, Navajas R, Maranon C. et al. Incomplete tyrosine 1680 sulphation in recombinant FVIII concentrates. Haemophilia 2011; 17: 709-710.
    CrossrefPubMedGoogle Scholar
  • 11 Dasgupta S, Repesse Y, Bayry J. et al. VWF protects FVIII from endocytosis by dendritic cells and subsequent presentation to immune effectors. Blood 2007; 109: 610-612.
    CrossrefPubMedGoogle Scholar
  • 12 Qadura M, Waters B, Burnett E. et al. Recombinant and plasma-derived factor VIII products induce distinct splenic cytokine microenvironments in haemophilia A mice. Blood 2009; 114: 871-880.
    CrossrefPubMedGoogle Scholar
  • 13 Ghio M, Contini P, Ottonello L. et al. Effect of clotting factors concentrates on lymphocyte and neutrophil function in vitro. Thromb Haemost 2003; 89: 365-373.
    Article in Thieme ConnectPubMedGoogle Scholar
  • 14 Mannucci PM, Mancuso ME, Santagostino E. How we choose factor VIII to treat haemophilia. Blood 2012; 119: 4108-4114.
    CrossrefPubMedGoogle Scholar
  • 15 Marcucci M, Mancuso ME, Santagostino E, et al. Type and intensity of FVIII exposure on inhibitor development in PUPs with haemophilia A. A patient-level meta-analysis. Thromb Haemost 2015; 113: 958-967.
    Article in Thieme ConnectPubMedGoogle Scholar
  • 16 Mannucci PM, Gringeri A, Peyvandi F. et al. Factor VIII products and inhibitor development: the SIPPET study (survey of inhibitors in plasmaproduct exposed toddlers). Haemophilia 2007; 13 (Suppl. 05) 65-68.
    PubMedGoogle Scholar
  • 17 Gouw SC, van der Bom JG, Ljung R. et al. Factor VIII products and inhibitor development in severe haemophilia A. N Engl J Med 2013; 368: 231-239.
    CrossrefPubMedGoogle Scholar
  • 18 Iorio A, Skinner MW, Makris M. Factor VIII products and inhibitors in severe haemophilia A. N Engl J Med 2013; 368: 1456.
    CrossrefPubMedGoogle Scholar
  • 19 Kessler CM, Iorio A. The Rodin (Research Of Determinants of INhibitor Development among PUPs with haemophilia) study: the clinical conundrum from the perspective of haemophilia treaters. Haemophilia 2013; 19: 351-354.
    CrossrefPubMedGoogle Scholar
  • 20 Calvez T, Chambost H, Claeyssens-Donadel S. et al Recombinant factor VIII products and inhibitor development in previously untreated boys with severe haemophilia A. Blood 2014; 124: 3398-3408.
    CrossrefPubMedGoogle Scholar
  • 21 Collins PW, Palmer BP, Chalmers EA. et al. Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe haemophilia A, 2000–2011. Blood 2014; 124: 3389-3397.
    CrossrefPubMedGoogle Scholar
  • 22 Fischer K, Lassila R, Peyvandi F. et al. Inhibitor development in haemophilia according to concentrate. Four-year results from the European HAemophilia Safety Surveillance (EUHASS) project. Thromb Haemost 2015; 113: 968-975.
    Article in Thieme ConnectPubMedGoogle Scholar