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Hamostaseologie 2010; 30(S 01): S141-S145
DOI: 10.1055/s-0037-1619096
Communication
Schattauer GmbH

Inhibitors in children with severe haemophilia A treated with recombinant and plasmatic FVIII products

A hypothesis-based cumulative metaanalysisInhibitorentwicklung bei Kindern mit schwerer Hämophilie A unter Behandlung mit rekombinantem oder plasmatischem Faktor-VIII-Konzentrat
S. Halimeh
1   Medical Thrombosis and Haemophilia Treatment/Blood Transfusion Center Duisburg, Germany
,
C. Bidlingmaier
2   Department of Paediatrics, University Hospital Munich, Germany
,
L.R. Brandao
3   Department of Paediatric Haematology/Oncology, The Hospital for Sick Children, Toronto, Canada
,
C. Escuriola Ettingshausen
4   Department of Paediatric Haematology/Oncology, University Hospital Frankfurt, Germany
,
S. Holzhauer
5   Department of Paediatric Haematology/Oncology, Charité, Berlin, Germany
,
G. Kenet
6   The Israel National Haemophilia Centre, Sheba Medical Centre, Tel-Hashomer, Israel
,
R. Knöfler
7   Department of Paediatric Haematology/Oncology, University Hospital Dresden, Germany
,
W. Kreuz
4   Department of Paediatric Haematology/Oncology, University Hospital Frankfurt, Germany
,
K. Kurnik
2   Department of Paediatrics, University Hospital Munich, Germany
8   Department of Paediatric Haematology/Oncology, Univ. Hospital Münster, Germany
,
D. Manner
8   Department of Paediatric Haematology/Oncology, Univ. Hospital Münster, Germany
,
U. Nowak-Göttl
8   Department of Paediatric Haematology/Oncology, Univ. Hospital Münster, Germany
› Author Affiliations Funding sources The study was supported by grants from the Förderverein Schlaganfall und Thrombosen im Kindesalter e.V. These study supporters had no role on study design, data collection, data analysis, data interpretation or in writing of the report.
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Publication History

Publication Date:
23 December 2017 (online)

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  • References

  • 1 Addiego J, Kasper C, Abildgaard C. et al. Frequency of inhibitor development in haemophiliacs treated with low-purity factor VIII. Lancet 1993; 342: 462-464.
    CrossrefPubMedGoogle Scholar
  • 2 Astermark J, Petrini P, Tengborn L. et al. Primary prophylaxis in severe hemophilia should be started at an early age but can be individualized. Br J Haematol 1999; 105: 1109-1113.
    CrossrefPubMedGoogle Scholar
  • 3 Bidlingmaier C, Manner D, Halimeh S. et al. Influence of factor VIII products, viral inactivation and dosage regimens on meaningful inhibitor development in children with severe hemophilia A: Results of a non-concurrent cohort study. J Thromb Haemost. 2009 07. suppl. 02, PP-MO-607.
    PubMedGoogle Scholar
  • 4 Blanchette VS, Manco-Johnson M, Santagostino E. et al. Optimizing factor prophylaxis for the haemophilia population: where do we stand? Haemophilia. 2004; 10: 97-104.
    PubMedGoogle Scholar
  • 5 Bray GL, Gomperts S, Courter R. et al. A multicenter study of recombinant factor VIII (Recombinate): safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. Blood 1994; 83: 2428-2435.
    PubMedGoogle Scholar
  • 6 Briet E, Rosendaal FR, Kreuz W. et al. High titer inhibitors in severe haemophilia A. A meta-analysis based on eight long-term follow-up studies concerning inhibitors associated with crude or intermediate purity factor VIII products. Thromb Haemost 1994; 72: 162-164.
    Article in Thieme ConnectPubMedGoogle Scholar
  • 7 Calvez T, Laurian Y, Goudemand J. Inhibitor incidence with recombinant vs. Plasma-derived FVIII in previously untreated patients with severe hemophilia A: homogenous results from for published observational studies. J Thromb Haemost 2008; 06: 390-392.
    CrossrefPubMedGoogle Scholar
  • 8 Chalmers EA, Brown SA, Keeling D. et al. Early factor VIII exposure and subsequent inhibitor development in children with severe haemophilia A. Haemophilia 2007; 13: 149-155.
    CrossrefPubMedGoogle Scholar
  • 9 Courter SG, Bedrosian CL. Clinical evaluation of B-Domain deleted recombinant factor VIII in previously untreated patients. Sem Hematol 2001; 38: 52-59.
    CrossrefPubMedGoogle Scholar
  • 10 De Biasi R, Rocino A, Papa ML. et al. Incidence of factor VIII inhibitor development in hemophilia A patients treated with less pure plasma derived concentrates. Thromb Haemost 1994; 71: 544-547.
    Article in Thieme ConnectPubMedGoogle Scholar
  • 11 Ehrenforth S, Kreuz W, Scharrer I. et al. Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs. Lancet 1992; 339: 594-598.
    CrossrefPubMedGoogle Scholar
  • 12 Ehrlich HJ, Bray GL, Gomperts ED. Comparison of high responder inhibitor frequency in recent studies of previously untreated patients with hemophilia A. Thromb Haemost 1998; 79: 242-243.
    Article in Thieme ConnectPubMedGoogle Scholar
  • 13 Escuriola CEttingshausen, Kreuz W. Recombinant vs. plasma-derived products, especially those with intact vWF, regarding inhibitor development. Haemophilia 2006; 12: S102-S106.
    PubMedGoogle Scholar
  • 14 Fischer K, van der Bom JG, Mauser-Bunschoten EP. et al. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy. Haemophilia 2001; 07: 446-452.
    CrossrefPubMedGoogle Scholar
  • 15 Gill FM. The natural history of factor VIII inhibitors in patients with hemophilia A. In: Hoyer LW. Factor VIII inhibitors. New York: Alan R Liss; 1984: 19-29.
    Google Scholar
  • 16 Goudemand J, Rothschild C, Demiguel V. et al. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. Blood 2006; 107: 46-51.
    CrossrefPubMedGoogle Scholar
  • 17 Gouw SC, van der Bom JG, Auerswald G. et al. Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: the CANAL cohort study. Blood 2007; 109: 4693-4697.
    CrossrefPubMedGoogle Scholar
  • 18 Gringeri A, Monzini M, Tagariello G. et al. Occurrence of inhibitors in previously untreated or minimally treated patients with haemophilia A after exposure to a plasma-derived solvent-detergent factor VIII concentrate. Haemophilia 2006; 12: 128-132.
    PubMedGoogle Scholar
  • 19 Guerois C, Larian Y, Rothschild C. et al. Incidence of factor VIII inhibitor development in severe hemophilia A patients treated only with one brand of high purified plasma-derived concentrate. Thromb Haemost 1995; 73: 215-218.
    Article in Thieme ConnectPubMedGoogle Scholar
  • 20 Harbord RM, Egger M, Sterne JAC. A modified test for small-study effects in meta-analyses of controlled trials with binary endpoints. Satist Med 2006; 25: 3443-3457.
    PubMedGoogle Scholar
  • 21 Higgins JPT, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in meta-analysis. Br Med J 2003; 327: 557-560.
    CrossrefPubMedGoogle Scholar
  • 22 Iorio A, Halimeh S, Holzhauer S. et al. Rate of inhibitor development in previously-untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates. A systematic review. J Thromb Haemost 2010; 08: 1256-1265.
    CrossrefPubMedGoogle Scholar
  • 23 Kasper C. Complications of hemophilia A treatment: factor VIII inhibitors. Ann NY Acad Sci 1991; 614: 97-105.
    CrossrefPubMedGoogle Scholar
  • 24 Kirkwood BR, Sterne JAC. Medical statistics. Blackwell Science. 2nd ed.. 2003: 146-387.
    Google Scholar
  • 25 Kreuz W, Escuriola CEttingshausen, Zyschka A. et al. Inhibitor development in previously untreated patients with hemophilia A: A prospective longterm follow-up comparing plasma-derived and recombinant products. Sem Thromb Haemost 2002; 28: 285-290.
    Article in Thieme ConnectPubMedGoogle Scholar
  • 26 Kreuz W, Gill JC, Rothschild C. et al. Full-length sucrose-formulated recombinant factor VIII for treatment of previously untreated or minimally treated young children with severe haemophilia A. Thromb Haemost 2005; 93: 457-467.
    Article in Thieme ConnectPubMedGoogle Scholar
  • 27 Ljung R. Paediatric care of the child with haemophilia. Haemophilia 2002; 08: 178-182.
    CrossrefPubMedGoogle Scholar
  • 28 Lorenzo JI, Garcia R, Molina R. Factor VIII and IX inhibitors in haemophiliacs. Lancet 1992; 339: 1550-1551.
    CrossrefPubMedGoogle Scholar
  • 29 Lusher JM, Salzman PM. Viral safety and inhibitor development associated with factor VIIIC ultrapurified from plasma in hemophiliacs previously unexposed to factor VIIIc concentrates. The Monoclate Study Group. Semin Hematol 1990; 27: 1-7.
    PubMedGoogle Scholar
  • 30 Lusher JM, Arkin S, Abildgaard CF, Schwartz RS. Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. New Engl J Med 1993; 328: 453-459.
    CrossrefPubMedGoogle Scholar
  • 31 Mayer D. Essential evidence-based medicine. Cambridge University Press; 2004: 347-360.
    Google Scholar
  • 32 Morado M, Villar A, Jimenez VYuste. et al. Prophylactic treatment effects on inhibitor risk: experience in one centre. Haemophilia 2005; 11: 79-83.
    CrossrefPubMedGoogle Scholar
  • 33 Nilsson IM, Berntorp E, Löfqvist T. et al. Twentyfive years’experience of prophylactic treatment in severe hemophilia A and B. J Intern Med 1992; 232: 25-32.
    CrossrefPubMedGoogle Scholar
  • 34 Peerlinck K, Rosendaal FR, Vermylen J. Incidence of inhibitor development in a group of young hemophilia patients treated exclusively with lyophilized cryoprecipitate. Blood 1993; 81: 3332-3335.
    PubMedGoogle Scholar
  • 35 Rasi V, Ikkala E. Haemophiliacs with factor VIII in Finland: prevalence, incidence and outcome. Br J Haematol 1990; 76: 369-371.
    CrossrefPubMedGoogle Scholar
  • 36 Rothschild C, Laurian Y, Satre EP. et al. French previously untreated patients with severe hemophilia A after exposure to recombinant factor VIII: Incidence of inhibitor and evaluation of immune tolerance. Thromb Haemost 1998; 80: 779-783.
    Article in Thieme ConnectPubMedGoogle Scholar
  • 37 Schwarts RS, Abildgaard CF, Aledort LM. et al. Human recombinant DNA-derived antihemophilic factor (FVIII) in the treatment of hemophilia A. N Engl J Med 1990; 323: 1800-1805.
    CrossrefPubMedGoogle Scholar
  • 38 Schwarzinger I, Pabinger I, Korninger C. et al. Incidence of inhibitors in patients with severe and moderate hemophilia A treated with factor VIII concentrates. Am J Hematol 1987; 24: 241-245.
    CrossrefPubMedGoogle Scholar
  • 39 Strauss T, Ravid B, Martinowitz U. et al. Early exposure to recombinant factor concentrates may increase inhibitor development: a single center cohort study. Haemophilia 2008; 14 (02) 52.
    CrossrefPubMedGoogle Scholar
  • 40 Vandenbroucke JP, von Elm E, Altman DG. et al. Strengthening the reporting of observational studies in epidemiology (STROBE): Explanation and elaboration. Ann Intern Med 2007; 147: W163-W194.
    PubMedGoogle Scholar
  • 41 Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003; 09: 418-435.
    CrossrefPubMedGoogle Scholar
  • 42 Yee TT, Williams MD, Hill FGH. et al. Absence of inhibitors in previously untreated patients with severe haemophilia A after exposure to a singe intermediate purity factor VIII product. Thromb Haemost 1997; 78: 1027-1029.
    Article in Thieme ConnectPubMedGoogle Scholar