Use of recombinant factor VIIa in 3 patients with inherited type I Glanzmann's thrombasthenia undergoing invasive procedures

• Published in: Thrombosis and haemostasis Vol. 83; no. 5; p. 644
• Main Authors: d'Oiron, R, Ménart, C, Trzeciak, M C, Nurden, P, Fressinaud, E, Dreyfus, M, Laurian, Y, Négrier, C
• Format: Journal Article
• Language: English
• Published: Germany 01.05.2000
• Subjects: Adenocarcinoma - complications; Adenocarcinoma - surgery; Adult; Aged; Cholecystectomy; Colectomy; Colonic Neoplasms - complications; Colonic Neoplasms - surgery; Factor VIIa - therapeutic use; Female; Genes, Recessive; Humans; Laparotomy; Male; Middle Aged; Platelet Glycoprotein GPIIb-IIIa Complex - immunology; Recombinant Proteins - therapeutic use; Thrombasthenia - complications; Thrombasthenia - drug therapy
• ISSN: 0340-6245
• DOI: 10.1055/s-0037-1613884

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 microg/kg) was immediately followed by continuous infusion at the rate of 9-30 microg/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.