Off-label use of rFVIIa in children with excessive bleeding: a consecutive study of 153 off-label uses in 139 children.
BACKGROUND
Recombinant factor VIIa is a general hemostatic agent. Randomized trials have demonstrated effectiveness in adults; however, data in children are confined to case reports and series subject to publication bias.


METHODS
A consecutive cohort of children treated with rFVIIa was identified via inspection of pharmacy records. Data collected included demographic data, underlying disorders, reason for rFVIIa use, dosing, thrombotic events, and mortality. Efficacy was scored subjectively on a 3-point scale (complete, partial, or no response) and supported by a measurement of transfusion of red cells, platelets, fresh frozen plasma, and cryoprecipitate in the 3 days prior to and following use of rFVIIa. Patients with hemophilia and congenital factor VII deficiency were excluded.


RESULTS
Sixty-five percent had a complete response and 26% had a partial response. Following treatment, there was a mean reduction in transfusions of 14.2 ml/kg for red blood cells, 10.9 ml/kg for platelets, 9 ml/kg for fresh frozen plasma, and 4.6 ml/kg for cryoprecipitate. Thrombosis occurred in 4.3% of patients with the highest rate being in neonates (17.6%).


CONCLUSIONS
rFVIIa is an effective general hemostatic agent for management of excessive bleeding in children and that with the exception of neonates has excellent safety.
Children's Hospital of Orange County, Orange, California, USA. gyoung@chla.usc.edu
Guy
G
Young
Brian
B
Wicklund
Philip
P
Neff
Chelsea
C
Johnson
Diane J
DJ
Nugent
D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D015942:Factor VIIa / Q000008:administration & dosage* / Q000009:adverse effects; D006470:Hemorrhage / Q000188:drug therapy* / Q000401:mortality; D006490:Hemostatics / Q000008:administration & dosage* / Q000009:adverse effects; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000009:adverse effects; D013927:Thrombosis / Q000139:chemically induced
D006490:Hemostatics; D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa