Prophylactic and therapeutic recombinant factor VIIa administration to patients with Glanzmann's thrombasthenia: Results of an international survey

Man Chiu Poon*, R. d'Oiron, M. von Depka, K. Khair, C. Négrier, A. Karafoulidou, A. Huth-Kuehne, M. Morfini, A. Street, S. Kuhle, C. van Geet, J. Wu, C. Demers, D. S. Houston, V. Blanchette, T. Lambert, L. de Lumley, E. Fressinaud, A. Marquès-Verdier, C. BergerP. Beurrier, P. Paugy, B. Pautard, M. F. Torchet, N. Trillot, M. Vicariot, H. Chambost, V. Guérin, P. Sié, C. Guethner, K. Kurnik, P. E. Makris, G. Kenet, R. Musso, J. Lorenzo, P. Petrini, L. Tengborn, S. Lethagen, W. B.J. Gerrits, O. Devecioglu, A. Thomas, J. Wilde, M. Winter, H. A. Britton, T. Abshire, M. Heisel, A. Shapiro, I. Warrier, W. Y. Wong, A. Chuansumrit

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review


Background: Antibodies to glycoprotein (GP) IIb-IIIa and/or HLA may render platelet transfusions ineffective to stop bleeding or to cover surgery in patients with Glanzmann's thrombasthenia (GT). Anecdotal reports suggest recombinant factor (rF)VIIa might be a therapeutic alternative in these situations. Objectives: An international survey was conducted to evaluate further the efficacy and safety of rFVIIa in GT patients. Patients: We analyzed the use of rFVIIa during 34 surgical/invasive procedures and 108 bleeding episodes in 59 GT patients including 29 with current or previous antiplatelet antibodies, and 23 with a history of refractoriness to platelet transfusion. Results: rFVIIa was effective in 29 of the 31 evaluable procedures, and in 77 of the 103 evaluable bleeding episodes of which eight had a recurrence. A significantly higher success rate was observed in severe bleeding episodes when an arbitrarily defined 'optimal regimen' derived from the Canadian pilot study results (≥ 80 μg kg-1 rFVIIa/injection, dosing interval ≤2.5 h, three or more doses before failure declaration) was used compared with other regimens (77%; 24/31 vs. 48%, 19/40; χ2, P = 0.010). Patients given maintenance doses had significantly fewer recurrences within 48 h of bleed cessation compared with those not given any (Fisher's exact test, P = 0.022). One thromboembolic event and one blood clot in the ureter occurring in surgical patients following prolonged continuous infusion of high-dose rFVIIa and antifibrinolytic drug use have been previously reported. Conclusion: rFVIIa seems a potential alternative to platelet transfusion in GT patients, particularly in those with antiplatelet antibodies and/or platelet refractoriness.

Original languageEnglish
Pages (from-to)1096-1103
Number of pages8
JournalJournal of Thrombosis and Haemostasis
Issue number7
StatePublished - Jul 2004


  • Antibodies to glycoprotein IIb-IIIa
  • Glanzmann's thrombasthenia
  • Platelet transfusion
  • Recombinant factor VIIa


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