Sixteen Years of Treatment with Pasteurized Human Clotting Factor Concentrates in Children and Adolescents: A Pharmacosurveillance Investigation Comprising 727 Patient Years

Wolfgang Muntean; Werner Zenz; Jutta Falger; Christoph Male; Werner Streif; Johann Wank; Rudolf Schwarz; Klaus Schmitt; Peter Kurnik; Karl Zwiauer

doi:10.1055/s-2002-30198

Seminars in Thrombosis and Hemostasis, Table of Contents

Semin Thromb Hemost 2002; 28(S1): 063-074
DOI: 10.1055/s-2002-30198

Sixteen Years of Treatment with Pasteurized Human Clotting Factor Concentrates in Children and Adolescents: A Pharmacosurveillance Investigation Comprising 727 Patient Years

Wolfgang Muntean^1, ² , Werner Zenz^1, ² , Jutta Falger³ , Christoph Male³ , Werner Streif⁴ , Johann Wank⁵ , Rudolf Schwarz⁶ , Klaus Schmitt⁶ , Peter Kurnik⁷ , Karl Zwiauer⁸

¹Department of Pediatrics, University of Graz, Austria
²Ludwig Bolzmann Research Institute for Pediatric Hemostasis and Thrombosis, Graz, Austria
³Department of Pediatrics, University of Vienna, Austria
⁴Department of Pediatrics, University of Innsbruck, Austria
⁵St. Anna Children Hospital, Vienna, Austria
⁶Children Hospital, Linz, Austria
⁷Children Hospital, Klagenfurt, Austria
⁸Children Hospital, St. Pölten, Austria

Abstract

Full Text

References

REFERENCES

1 Mannucci P M. Modern treatment of hemophilia: from the shadows towards the light. Thromb Haemost . 1993; 70 17-23
2 Teitel J M. Safety of coagulation factor concentrates. Haemophilia . 1998; 4 393-401
3 Ludlam CA.Viral safety of plasma-derived factor VIII und IX concentrates. Blood Coagul Fibrinolysis . 1997; 8 19-23
4 Schimpf K, Brackmann H H, Kreuz W. Absence of antihuman immunodeficiency virus types I and II seroconversion after the treatment of haemophilia A or von Willebrand disease with pasteurized factor VIII concentrate N Engl J Med . 1989; 321 1148-1151
5 Mannucci P M, Schimpf K, Brettler D B. Low risk for hepatitis C in hemophiliacs given a high-purity, pasteurized factor VIII concentrate. Ann Intern Med . 1990; 113 27-32
6 Zenz W, Hoffmann H, Muntean W. Hepatitis C-Antikörper bei Kindern und Jugendlichen mit angeborenen Gerinnungsstörungen. Wien Klin Wochenschr . 1991; 3 69-72
7 Zakrzewska K, Azzi A, Patou G. Human parvovirus B 19 in clotting factor concentrates: B 19 DNA detection by the nested polymerase chain reaction. Br J Haematol . 1992; 81 407-412
8 Azzi A, Ciappi S, Zakvrzewska K. Human parvovirus B19 infection in hemophiliacs first transfused with two high-purity, virally attenuated factor VIII concentrates. Am J Hematol . 1992; 39 228-230
9 Eis-Hübinger A M, Sasowski U, Brackmann H H. Parvovirus B19 is frequently present in recombinant coagulation factor VIII products. Thromb Haemost (Letter) . 1996; 76 1120
10 Flores G, Juarez J C, Montoro J B. Seroprevalence of parvovirus B19, cytomegalovirus, hepatitis A virus and hepatitis E virus antibodies in hemophiliacs treated exclusively with clotting-factor concentrates considered safe against human immunodeficiency and hepatitis C viruses. Hemophilia . 1995; 1 115-117
11 Gilles J GG, Marc C, Saint-Remy J MR. Factor VIII inhibitors. Thromb Haemost . 1997; 78 641-646
12 Bray G L, Gomperts E, Courter S. A multicenter study of recombinant factor VIII (Recombinante^℗): safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. Blood . 1994; 83 2428-2435
13 Addiego J, Kasper C, Abildgaard C. Frequency of inhibitor development in haemophiliacs treated with low-purity factor VIII. Lancet . 1993; 342 462-464
14 De Brasi R, Rocino A, Papa M L. Incidence of factor VIII inhibitor development in hemopilia A patients treated with less pure plasma derived concentrates. Thromb Haemost . 1994; 71 544-547
15 Scharrer I, Neutzling O. Incidence of inhibitors in haemophiliacs. A review of the literature. Blood Coagul Fibrinolysis . 1993; 4 753-758
16 McMillan C W, Shapiro S S, Whitehurst D. The natural history of factor VIII inhibitors in patients with hemophilia A: a national cooperative study. II. Observations on the initial development of factor VIII:C inhibitors. Blood . 1988; 71 344-348
17 Peerlinck K, Arnout J, Gilles J G, Saint-Remy J M, Vermylen J. A higher than expected incidence of factor VIII inhibitors in multitransfused haemophilia A patients treated with an intermediate purity pasteurized factor VIII concentrate. Thromb Haemost . 1993; 69 115-118
18 Rosendaal F, Nieuwenhuis H K, van den Berg M H. A sudden increase in factor VIII inhibitor development in multitransfused hemophilia A patients in The Netherlands. Blood . 1993; 81 2180-2186
19 Lusher J M. Viral safety and inhibitor development associated with monoclonal antibody-purified FVIIIC. Ann Hematol . 1991; 63 138-141
20 Guerois C, Laurian Y, Rothschild C. Incidence of factor VIII inhibitor development in severe hemophilia A patients treated only with one brand of highly purified plasma-derived concentrate. Thromb Haemost . 1995; 73 215-218
21 Mannucci P M, Brettler D B, Aledort L M. Immune status of human immunodeficiency virus seropositive and seronegative hemophiliacs infused for 3.5 years with recombinant factor VIII. Blood . 1994; 83 1958-1962
22 Allersma D P, Smid W M, van der Does A J, van der Meer J, Briet E. Effects of chronic factor VIII substitution on immune parameters in HIV seronegative haemophiliacs: a comparison between cryoprecipitate and factor VIII concentrate. Thromb Haemost . 1996; 75 261-266
23 Shannon B T, Roach J, Cheek-Luten M, Orosz C, Ruymann F B. Progressive change in lymphocyte distribution and degree of hypergammaglobulinemia with age in children with hemophilia. J Clin Immunol . 1986; 6 121-129
24 Hassett J, Gjerset G F, Mosley J W. Effect on lymphocyte subsets of clotting factor therapy in human immunodeficiency virus-1-negative congenital clotting disorders. Blood . 1993; 82 1351-1357
25 Funk M, Ebener U, Kreuz W. Immune status of HIV and HCV negative haemophiliacs treated with intermediate-purity factor VIII concentrates. Lancet . 1993; 342 933-934
26 Varon D, Schulman S, Dardik R. High versus ultra-high purity factor VIII concentrate therapy: prospective evaluation of immunological and clinical parameters in HIV seronegative and seropositive hemophiliacs. Thromb Haemost . 1994; 72 359-362
27 Schulman S. Effects of factor VIII concentrates on the immune system in hemophilic patients. Ann Hematol . 1991; 63 145-151
28 Glöckel U, Gromball J, Kobatsch B. Influence of factor VIII, factor IX and immunoglobulin preparations on proliferative responses of mitogen-activated lymphocytes. Immunol Infect . 1987; 15 62-65
29 De Biasi R, Rocino A, Miraglia E, Mastruelle L, Quirino A A. 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 1919-1922
30 Hilgartner M W, Buckley J D, Operskalski E A, Pike M C, Mosley J W. Transfusion Safety Study Group. Purity of factor VIII concentrates and serial CD4 counts. Lancet . 1993; 341 1373-1374
31 Kasper C K, Aledort L M, Aronson D. Proceedings: a more uniform measurement of factor VIII inhibitors. Thrombosis et Diathesis Haemorrhagica (Abst) . 1975; 34 612
32 Neumann K. The signal strengthening power of statistics: a demonstration of regression with unknown model domain in statistics and pharmacoeconomics. Pharmacoepidemiol Drug Safety . 1997; 6 27-29
33 Thomas L, ed. . Labor und Diagnose Marburg: Die Medizinische Verlagsgesellschaft; 1992
34 Commans-Bitter W M, De Grool R, van den Beernd R. Immunophenotyping of blood lymphocytes in childhood. Reference values for lymphocyte subpopulations. J Pediatr . 1997; 130 388-393
35 Mannucci P M, Gdovin S, Gringeri A. Transmission of hepatitis A to patients with hemophilia by factor VIII concentrates treated with organic solvent and detergent to inactivate viruses. The Italian Collaborative Group. Ann Intern Med . 1994; 120 1-7
36 Prodinger W M, Larcher C, Solder B M, Geissler D, Dierich M P. Hepatitis A in western Austria, the epidemiological situation before the introduction of active immunisation. Infection . 1994; 22 53-55
37 Kerr J R. Parvovirus B19 infection. Eur J Clin Microbiol Infect Dis . 1996; 15 10-29
38 Eis-Hubinger A M, Oldenburg J, Brackmann H H, Matz B, Schneweis K E. The prevalence of antibody to parvovirus B19 in hemophiliacs and in the general population. Zentralbl Bakteriol . 1996; 284 232-240
39 Santagostino E, Mannucci P M, Gringeri A. Transmission of parvovirus B19 by coagulation factor concentrates exposed to 100 degrees C heat after lyophilization. Transfusion . 1997; 37 517-522
40 Aygören-Pürsün E, Scharrer I, and the German Kogenate Study Group. A multicenter pharmacosurveillance study for the evaluation of the efficacy and safety of recombinant factor VIII in the treatment of patients with hemophilia A. Thromb Haemost . 1997; 78 1352-1356
41 Morfini M, Longo G, Rossi Ferrini P. Hypoplastic anemia in a hemophiliac first infused with a solvent/detergent treated factor VIII concentrate: the role of human B19 parvovirus. Am J Hematol . 1992; 39 149-150
42 Yee T T, Lee C A, Pasi K J. Life-threatening human parvovirus B19 infection in immunocompetent hemophilia. Lancet . 1995; 345 794-795
43 Briet E, Rosendaal F R, Kreuz W. 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
44 Scandella D, Mondorf W, Klinge J. The natural history of the immune response to exogenous factor VIII in severe haemophilia A. Haemophilia . 1998; 4 546-551
45 Lechner K, Niessner H, Bettelheim P. T-cell alterations in hemophiliacs treated with commercial clotting factor concentrates. Thromb Haemost . 1983; 50 552-556