Hemmkörperentwicklung bei Hämophilie-Patienten nach Präparate wechsel

Summary

The retrospective observational study surveys the relationship between development of inhibitors in the treatment of haemophilia patients and risk factors such as changing FVIII products. A total of 119 patients were included in this study, 198 changes of FVIII products were evaluated. Results: During the observation period of 12 months none of the patients developed an inhibitor, which was temporally associated with a change of FVIII products. A frequent change of FVIII products didn’t lead to an increase in inhibitor risk. The change between plasmatic and recombinant preparations could not be confirmed as a risk factor. Furthermore, no correlation between treatment regimens, severity, patient age and comorbidities of the patients could be found.

Zusammenfassung

Die retrospektive Beobachtungsstudie untersucht den Zusammenhang zwischen Hemmkörperbildung in der Therapie von Hämophilie-Patienten und Risikofaktoren wie häufigem Präparatewechsel. Insgesamt nahmen 119 Patienten an der Studie teil, es wurden 198 Präparatewechsel ausgewertet. Ergebnisse: Während des Beobachtungszeitraums von 12 Monaten entwickelte keiner der Patienten einen Hemmkörper, der in zeitlichem Zusammenhang mit einer Umstellung stand. Ein häufiger Wechsel führte zu keiner Erhöhung des Hemmkörperrisikos. Auch der Wechsel zwischen plasmatisch und rekombinant hergestellten Präparaten konnte nicht als Risikofaktor bestätigt werden. Des Weiteren fand sich kein Zusammenhang zwischen Therapieregime, Schweregrad, Patientenalter und Komorbiditäten der Patienten.

Keywords

Haemophilia A - inhibitor - change of FVIII product

Schlüsselwörter

Hämophilie - Hemmkörper - Präparatewechsel

• Literatur

• 1 Astermark J. Basic aspects of inhibitors to factors VIII an IX and the influence of non-genetic risk factors. Haemophilia 2006; 12 (06) 8-13.
• 2 Gouw SC, van den Berg HM. The multifactorial etiology of inhibitor development in hemophilia: genetics and environment. Semin Thromb Haemost 2009; 35: 723-734.
• 3 Gouw SC, van der Bom JG M, van den Berg H. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood 2007; 109: 4648-4654.
• 4 Mc CWMillan, Shapiro SS, Whitehurst D. et al. The natural history of factor VIII:C 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.
• 5 White GC, DiMichele DM, Mertens K. et al. Utilisation of previously untreated patients (PTPs), non infected patients (NIPs), and previously untreated patients (PUPs) in the evaluation of new factor VIII and factor IX concentrates. Thromb Haemost 1999; 81: 462.
• 6 Kempton CL, Soucie JM, Abshire TC. Incidence of inhibitors in a cohort of 838 males with hemophilia Apreviously treated with factor VIII concentrates. J Thromb Haemost 2006; 04: 2476-2481.
• 7 Lorenzo JI, Lopez A, Altisent C, Aznar JA. Incidence of factor VIII inhibitors in severe hemophilia: the importance of patient age. Br J Haematol 2001; 113: 600-603.
• 8 Van der Bom JG, Mauser-Bunschoten EP, Fischer K, van den Berg HM. Age at first treatment and immune tolerance to factor VIII in severe hemophilia. Thromb Haemost 2003; 89: 475-479.
• 9 Colvin BT, Hay CR, Hill FG, Preston FE. The incidence of factor VIII inhibitors in the United Kingdom, 1990–93. Inhibitor Working Party. Br J Haematol 1995; 89: 908-910.
• 10 Peerlinck K, Arnout J, Gilles JG. et al. 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.
• 11 Rosendaal FR, Nieuwenhuis HK, van den Berg HM. et al. A sudden increase in factor VIII inhibitor development in multitransfused haemophilia A patients in The Netherlands. Dutch Hemophilia Study Group. Blood 1993; 81: 2180-2186.
• 12 Bacon CL, Singleton E, Brady BB. et al. Low risk of inhibitor formation in haemophilia A patients following en masse switch in treatment to a third-generation full length plasma and albumin-free recombinant factor VIII producht (ADVATE®). Haemophilia 2011; 17: 407-411.
• 13 Hay CRM, Palmer B, Chalmers E. et al. Incidence of factor VIII inhibitors throughout life in severe hemophilia A in the United Kingdom. Blood 2011; 117: 6367-6370.
• 14 Giles AR, Rivard GE, Teiltel J. et al. Surveillance for factor VIII inhibitor development in the Canadian hemophilia A population following the widespread introduction of recombinant factor VIII replacement therapy. Transfus Sci 1998; 19: 139-148.
• 15 Rubinger M, Lillicrap D, Rivard GE. et al. A prospective surveillance study of factor VIII inhibitor development in the Canadian haemophilia A population following the switch to a recombinant factor VIII product formulated with sucrose. Haemophilia 2008; 14: 281-286.
• 16 Rosendaal FR, Nieuwnhuis HK, van den Berg HM. et al. A sudden increase in factor VIII inhibitor development in multitransfused hemophilia A patients in The Netherlands. Dutch Hemophilia Study Group. Blood 1993; 81: 2180-2186.