Use of Recombinant Factor VIIa in 3 Patients with Inherited Type I Glanzmann’s Thrombasthenia Undergoing Invasive Procedures

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.

Key words

Recombinant factor VIIa - Glanzmann’s thrombasthenia - anti-glycoprotein IIb-IIIa antibodies

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