Use of recombinant factor VIIa in 3 patients with inherited type I Glanzmann's thrombasthenia undergoing invasive procedures.
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.
d'Oiron
R
R
The Haemophilia Center, Hôpital Bicêtre, Paris Sud, France. rdoiron@calva.net
Ménart
C
C
Trzeciak
M C
MC
Nurden
P
P
Fressinaud
E
E
Dreyfus
M
M
Laurian
Y
Y
Négrier
C
C
D000230:Adenocarcinoma / Q000150:complications / Q000601:surgery; D000328:Adult; D000368:Aged; D002763:Cholecystectomy; D003082:Colectomy; D003110:Colonic Neoplasms / Q000150:complications / Q000601:surgery; D015942:Factor VIIa / Q000627:therapeutic use*; D005260:Female; D005808:Genes, Recessive; D006801:Humans; D007813:Laparotomy; D008297:Male; D008875:Middle Aged; D019039:Platelet Glycoprotein GPIIb-IIIa Complex / Q000276:immunology; D011994:Recombinant Proteins / Q000627:therapeutic use; D013915:Thrombasthenia / Q000150:complications / Q000188:drug therapy*
D019039:Platelet Glycoprotein GPIIb-IIIa Complex; D011994:Recombinant Proteins; D015942:Factor VIIa