Off-label use of rFVIIa in children with excessive bleeding: A consecutive study of 153 off-label uses in 139 children

• Published in: Pediatric blood & cancer Vol. 53; no. 2; pp. 179 - 183
• Main Authors: Young, Guy, Wicklund, Brian, Neff, Philip, Johnson, Chelsea, Nugent, Diane J.
• Format: Journal Article
• Language: English
• Published: Hoboken Wiley Subscription Services, Inc., A Wiley Company 01.08.2009
• Subjects: Child, Preschool; Dose-Response Relationship, Drug; Factor VIIa - administration & dosage; Factor VIIa - adverse effects; hemorrhage; Hemorrhage - drug therapy; Hemorrhage - mortality; Hemostatics - administration & dosage; Hemostatics - adverse effects; Humans; Infant; Infant, Newborn; Recombinant Proteins - administration & dosage; Recombinant Proteins - adverse effects; rFVIIa; thrombosis; Thrombosis - chemically induced
• ISSN: 1545-5009; 1545-5017; 1545-5017
• DOI: 10.1002/pbc.22053

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. Procedures 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. Pediatr Blood Cancer 2009;53:179–183. © 2009 Wiley‐Liss, Inc.