Off-label use of recombinant factor VIIa in pediatric patients

Pediatrics. 2012 Jun;129(6):e1533-40. doi: 10.1542/peds.2011-2561. Epub 2012 May 28.


Objective: To examine off-label recombinant factor VIIa (rFVIIa) use in pediatric patients including clinical indications, dose, adverse events, and outcomes.

Methods: All pediatric patients entered into the Haemostasis Registry from 75 participating hospitals were analyzed.

Results: Three hundred and eighty-eight pediatric patients received off-label rFVIIa from 2003 to 2009. Median age was 12 months (interquartile range 1 month to 11 years). Clinical context included cardiac surgery (52.1%), medical (11.6%), other surgery (10.8%), hematology/oncology (10.3%), trauma (9.3%), intracranial hemorrhage (3.1%), and liver disease (2.8%). Twenty-six patients received extracorporeal membrane oxygenation at the time of rFVIIa administration. Median first dose was 114 μg/kg (interquartile range 90-181; range 7-2250). Thirty-four percent received >1 dose. There was a reduction in usage of red blood cells, platelets, fresh-frozen plasma, and cryoprecipitate in the 24 hours after the first dose for all patients (all P values < .001). Thromboembolic adverse events (TEAs) were reported in 5.4%. No association between TEA and size of first dose was found. Where data were available, 82% of patients were subjectively classified as responding to rFVIIa. Overall 28-day mortality was 27%. In multivariate analysis, pH values before administration and clinical context were independently associated with response to first dose and 28-day mortality.

Conclusions: There was a significant reduction in blood product administration after rFVIIa and a subjective response rate of 82%. Both pH and clinical context were associated with response to rFVIIa and mortality. Overall, 5.4% had a TEA reported.

Publication types

  • Comparative Study
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Cohort Studies
  • Dose-Response Relationship, Drug
  • Extracorporeal Membrane Oxygenation
  • Factor VIIa / therapeutic use*
  • Female
  • Humans
  • Infant
  • Male
  • Off-Label Use*
  • Recombinant Proteins / blood
  • Recombinant Proteins / therapeutic use
  • Registries
  • Thromboembolism / blood
  • Thromboembolism / prevention & control


  • Recombinant Proteins
  • recombinant FVIIa
  • Factor VIIa