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

S. Halimeh; C. Bidlingmaier; L.R. Brandao; C. Escuriola Ettingshausen; S. Holzhauer; G. Kenet; R. Knöfler; W. Kreuz; K. Kurnik; D. Manner; U. Nowak-Göttl

doi:10.1055/s-0037-1619096

Hämostaseologie, Table of Contents

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

Authors

S. Halimeh

¹Medical Thrombosis and Haemophilia Treatment/Blood Transfusion Center Duisburg, Germany
C. Bidlingmaier

²Department of Paediatrics, University Hospital Munich, Germany
L.R. Brandao

³Department of Paediatric Haematology/Oncology, The Hospital for Sick Children, Toronto, Canada
C. Escuriola Ettingshausen

⁴Department of Paediatric Haematology/Oncology, University Hospital Frankfurt, Germany
S. Holzhauer

⁵Department of Paediatric Haematology/Oncology, Charité, Berlin, Germany
G. Kenet

⁶The Israel National Haemophilia Centre, Sheba Medical Centre, Tel-Hashomer, Israel
R. Knöfler

⁷Department of Paediatric Haematology/Oncology, University Hospital Dresden, Germany
W. Kreuz

⁴Department of Paediatric Haematology/Oncology, University Hospital Frankfurt, Germany
K. Kurnik

²Department of Paediatrics, University Hospital Munich, Germany

⁸Department of Paediatric Haematology/Oncology, Univ. Hospital Münster, Germany
D. Manner

⁸Department of Paediatric Haematology/Oncology, Univ. Hospital Münster, Germany
U. Nowak-Göttl

⁸Department of Paediatric Haematology/Oncology, Univ. Hospital Münster, Germany

Abstract

Full Text

References

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.
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.
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.
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.
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.
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.
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.
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.
9 Courter SG, Bedrosian CL. Clinical evaluation of B-Domain deleted recombinant factor VIII in previously untreated patients. Sem Hematol 2001; 38: 52-59.
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.
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.
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.
13 Escuriola CEttingshausen, Kreuz W. Recombinant vs. plasma-derived products, especially those with intact vWF, regarding inhibitor development. Haemophilia 2006; 12: S102-S106.
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.
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.
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.
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.
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.
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.
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.
21 Higgins JPT, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in meta-analysis. Br Med J 2003; 327: 557-560.
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.
23 Kasper C. Complications of hemophilia A treatment: factor VIII inhibitors. Ann NY Acad Sci 1991; 614: 97-105.
24 Kirkwood BR, Sterne JAC. Medical statistics. Blackwell Science. 2nd ed.. 2003: 146-387.
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.
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.
27 Ljung R. Paediatric care of the child with haemophilia. Haemophilia 2002; 08: 178-182.
28 Lorenzo JI, Garcia R, Molina R. Factor VIII and IX inhibitors in haemophiliacs. Lancet 1992; 339: 1550-1551.
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.
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.
31 Mayer D. Essential evidence-based medicine. Cambridge University Press; 2004: 347-360.
32 Morado M, Villar A, Jimenez VYuste. et al. Prophylactic treatment effects on inhibitor risk: experience in one centre. Haemophilia 2005; 11: 79-83.
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.
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.
35 Rasi V, Ikkala E. Haemophiliacs with factor VIII in Finland: prevalence, incidence and outcome. Br J Haematol 1990; 76: 369-371.
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.
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.
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.
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.
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.
41 Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003; 09: 418-435.
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.