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

J Thromb Haemost. 2004 Jul;2(7):1096-103. doi: 10.1111/j.1538-7836.2004.00767.x.

Abstract

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 (> or = 80 micro g kg(-1) rFVIIa/injection, dosing interval < or = 2.5 h, three or more doses before failure declaration) was used compared with other regimens (77%; 24/31 vs. 48%, 19/40; chi(2), 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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Child
  • Child, Preschool
  • Data Collection
  • Drug Evaluation
  • Factor VII / administration & dosage
  • Factor VII / therapeutic use*
  • Factor VIIa
  • Female
  • Hemorrhage / etiology
  • Hemorrhage / pathology
  • Humans
  • Infant
  • International Cooperation
  • Male
  • Middle Aged
  • Premedication
  • Recombinant Proteins / administration & dosage
  • Recombinant Proteins / therapeutic use*
  • Surgical Procedures, Operative / adverse effects
  • Thrombasthenia / complications
  • Thrombasthenia / drug therapy*

Substances

  • Recombinant Proteins
  • Factor VII
  • recombinant FVIIa
  • Factor VIIa