Subscribe to RSS
Download PDF
DOI: 10.1055/s-0037-1613884
Use of Recombinant Factor VIIa in 3 Patients with Inherited Type I Glanzmann’s Thrombasthenia Undergoing Invasive Procedures
Publication History
Received
14 June 1999
Accepted after resubmission
13 January 2000
Publication Date:
08 December 2017 (online)
Summary
The treatment of bleeds in Glanzmann’s thrombasthenia is a challenging issue, especially when repeated platelet transfusions have induced anti-glycoprotein (GP) IIb-IIIa or anti-HLA allo-immunisation. In an attempt to find an alternative treatment regimen, we used recombinant factor VIIa (rFVIIa, NovoSeven®, Novo Nordisk, Denmark) as first-line therapy in 3 patients with Glanzmann’s thrombasthenia and anti-GPIIb-IIIa iso-antibodies who were scheduled for invasive procedures. The administration of an initial bolus dose of rFVIIa (70–110 µg/kg) was immediately followed by continuous infusion at the rate of 9-30 µg/kg/h for 3–15 days. The treatment resulted in an excellent clinical efficacy and tolerance in 2 cases. In the third patient, whereas efficacy was excellent at the surgical site, pharyngonasal bleeds of traumatic origin persisted for 10 days, and a severe thromboembolic complication occurred 5 days after discontinuation of rFVIIa. Complementary studies are needed for patients with congenital platelet disorders in order to evaluate the safety and the potential therapeutic place of rFVIIa treatment.
-
References
- 1 George JG, Nurden JN, Caen JP. Glanzmann’s thrombasthenia: the spectrum of clinical disease. Blood 1990; 75: 1383-95.
- 2 Di Michele DM, Hathaway WE. Use of DDAVP in inherited and acquired platelet dysfunction. Am J Hematol 1990; 33: 39-45.
- 3 Mannucci PM. Desmopressin (DDAVP) in the treatment of bleeding disorders: the first 20 years. Blood 1997; 90: 2515-21.
- 4 Rao AK, Ghosh S, Sum L, Yang X, Disa J, Pickens P, Polanski M. Mechanisms of platelet dysfunction and response to DDAVP in patients with congenital platelet defects. A double-blind placebo controlled trial. Thromb Haemost 1995; 74: 1071-8.
- 5 Leach M, Makris M, Hampton KK, Preston FE. Norethisterone therapy for bleeding due to gastrointestinal telangiectases in Glanzmann’s thrombastenia. Br J Haematol 1998; 100: 594-6.
- 6 Bisch FC, Bowen KJ, Hanson BS, Kudryk VL, Billman MA. Dental considerations for a Glanzmann’s thrombasthenia patient: case report. J Periondotol 1996; 67: 536-40.
- 7 Gjorstrup P, Watt R. Therapeutic protein A Immunoadsorption. A review. Transfus Sci 1990; 11: 281-302.
- 8 Uehlinger J, Button GR, McCarthy J, Foster A, Watt R, Aledort LM. Immunoadsorption for coagulation factor inhibitors. Transfusion 1991; 31: 265-9.
- 9 Hedner U. Factor VIIa in the treatment of haemophilia. Blood Coagulat Fibrinol 1990; 01: 307-17.
- 10 Ingerslev J, Freidman D, Gastineau D, Gilchrist G, Johnsson H, Lucas G, McPherson J, Preston E, Scheibel E, Schuman M. Major surgery in haemophilic patients with inhibitors using recombinant factor VIIa. Haemostasis 1996; 26 (Suppl. 01) 118-23.
- 11 Lusher J, Ingerslev J, Roberts H, Hedner U. Clinical experience with recombinant factor VIIa. Blood Coagulat Fibrinol 1998; 09: 119-28.
- 12 Roberts HR. Clinical experience with activated factor VII: focus on safety aspects. Blood Coagulat Fibrinol 1998; 09 (Suppl. 01) S115-118.
- 13 Key NS, Aledort LM, Beardsley D, Cooper HA, Davignon G, Ewenstein BM, Gilchrist GS, Gill JC, Glader B, Hoots WK, Kisker CT, Lusher JM, Rosenfield CG, Shapiro AD, Smith H, Taft E. Home treatment of mild to moderate bleeding episodes using recombinant factor VIIa (NovoSeven) in haemophiliacs with inhibitors. Thromb Haemost 1998; 80: 912-8.
- 14 Hay CRM, Negrier C, Ludlam CA. The treatment of bleeding in acquired haemophilia with recombinant factor VIIa: a multicentric study. Thromb Haemost 1997; 78: 1463-7.
- 15 Tengborn L, Petruson B. A patient with Glanzmann’s thrombasthenia and epistaxis successfully treated with recombinant factor VIIa. Thromb Haemost 1996; 75: 971-82.
- 16 Wielenga JJ, Siebel Y, van Buuren HR, Berends FJ, Schipperus MR, van Vliet HDDM, Kappers MC. Use of recombinant factor VIIa and HLA matched platelets to prevent bleeding during and after major surgery in a patient with Glanzmann’s thrombasthenia. Haemophilia 1998; 04: 299 [abstract].
- 17 Kristensen J, Killander A, Hippe E, Helleberg C, Ellegård J. et al. Clinical experience with recombinant factor VIIa in patients with thrombocytopenia. Haemostasis 1996; 26 (Suppl. 01) 159-65.
- 18 Peters M, Heijboer H. Treatment of a patient with Bernard-Soulier syndrome and recurrent nose bleeds with recombinant factor VIIa. Thromb Haemost 1998; 80: 352.
- 19 Schiavoni M, Valenzano E, Mangini F, Inchingolo F, Micelli M, Mascolo E, Ciavarella N, Erhardtsen E, Hedner U. Efficacy of recombinant factor VIIa in patients affected by von Willebrand disease type III with alloantibodies against von Willebrand factor in oral surgery. Thromb Haemost 1997; (Suppl): 84-5.
- 20 Fressinaud E, Sigaud-Fiks M, Le Boterff C, Piot B. Use of recombinant factor VIIa for dental extraction in a patient affected by platelet-type (pseudo-) von Willebrand disease. Haemophilia 1998; 04: 299 [abstract].
- 21 Kiefel V, Santoso S, Weisheit M, Mueller-Eckhardt C. Monoclonal antibody-specific immobilization of platelet antigens (MAIPA): a new tool for the identification of platelet-reactive antibodies. Blood 1987; 70: 1722-6.
- 22 Schulman S, Bech MJensen, Varon D, Keller N, Gitel S, Horozowski H, Heim M, Martinowitz U. Feasibility of using recombinant factor VIIa in continuous infusion. Thromb Haemost 1996; 75: 432-6.
- 23 Schulman S, d’Oiron R, Martinowtz U, Pasi J, Briquel ME, MauserBunschoten E, Morfini B, Ritchie B, Goudemand J, Llyod J, McPherson J, Négrier C, Peerlinck K, Petrini P, Tusell J. Experiences with continuous infusion of recombinant activated factor VII. Blood Coagulat Fibrin 1998; 09 (Suppl. 01) S97-101.
- 24 Monroe DM, Hoffman M, Oliver JA, Roberts HR. A possible mechanism of action of activated factor VII independent of tissue factor. Blood Coagulat Fibrinol 1998; 09 (Suppl. 01) S15-20.
- 25 Hoffman M, Monroe DM, Oliver JA, Roberts HR. Factors IXa and Xa play distinct roles in tissue factor dependent initiation of coagulation. Blood 1995; 86: 1794-801.
- 26 Monroe DM, Hoffman M, Oliver JA, Roberts HR. Platelet activity of high-dose factor VIIa is independent of tissue factor. Br J Haematol 1997; 99: 542-7.
- 27 Hoffman M, Monroe DM, Roberts HR. Activated factor VII activates factors IX and X on the surface of activated platelets: thoughts on the mechanism of action of high-dose activated factor VII. Blood Coagulat Fibrinol 1998; 09 (Suppl. 01) S61-65.