Off-label use of recombinant factor VIIa in pediatric patients.
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.
Transfusion Research Unit, Department of Epidemiology and Preventive Medicine, Monash University, Melbourne, Victoria, Australia. zoe.mcquilten@monash.edu
Zoe K
ZK
McQuilten
Chris
C
Barnes
Amanda
A
Zatta
Louise E
LE
Phillips
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D004305:Dose-Response Relationship, Drug; D015199:Extracorporeal Membrane Oxygenation; D015942:Factor VIIa / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D056687:Off-Label Use; D011994:Recombinant Proteins / Q000097:blood / Q000627:therapeutic use; D012042:Registries; D013923:Thromboembolism / Q000097:blood / Q000517:prevention & control
D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa