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.
- factor VIIa/administration and dosage
- factor VIIa/adverse effects
- APTT —
- activated partial thromboplastin time
- ECMO —
- extracorporeal membrane oxygenation
- FFP —
- fresh-frozen plasma
- INR —
- international normalized ratio
- IQR —
- interquartile range
- rFVIIa —
- recombinant factor VIIa
- TEA —
- thromboembolic adverse event
- Accepted February 15, 2012.
- Copyright © 2012 by the American Academy of Pediatrics