title: Safety and efficacy of recombinant activated factor VII in nonhemophilia children with severe or life-threatening bleeding: a report from the SeveNBleeP registry.
abstract: We evaluated efficacy and safety of recombinant activated factor VII (rFVIIa) in nonhemophilia children with life-threatening or severe bleeding. Using data from the SeveNBleeP registry, we analyzed demographic, clinical, laboratory, and treatment data for children who received rFVIIa to treat severe hemorrhage. The SeveNBleeP registry was international registry formed in 2005, to collect information on the use of rFVIIa in the off-label setting of severe bleeding in nonhemophilia patients. There were 191 patient records entered into this registry, of which 164 were validated. Of the 164 records, in 137 patient records, rFVIIa was used for treatment of bleeding episodes. Of these 137 treatment episodes, 42 were in neonates and infants under 1 year of age. Use of rFVIIa significantly improved laboratory parameters (prothrombin time, international normalized ratio, activated partial thromboplastin time, hematocrit), reduced estimated blood loss, and reduced requirements for blood products (packed red blood cells and fresh frozen plasma) in those more than 1 year of age. There was no significant reduction in requirements for blood products after rFVIIa administration in the neonates and infants, but there was a trend to lower frequency of FFP use after rFVIIa administration. There was one thromboembolic event in an infant that was related to administration of rFVIIa. No other serious adverse events were reported that were related to administration of rFVIIa. In nonhemophilia-associated bleeding in children, rFVIIa appears to be safe and efficacious in reducing estimated blood loss in children over 1 year of age, although its effectiveness in infants below 1 year of age was less clear.
authors.affiliation: aDepartment of Paediatric Haematology, Children's University Hospital Brno, Brno, Czech Republic bDepartment of Pediatrics, University of New Mexico, Albuquerque, New Mexico, USA cDepartment of Pediatrics, Royal Children's Hospital, University of Melbourne, Melbourne, Australia dInstitute of Biostatistics and Analyses, Masaryk University, Brno, Czech Republic.
authors.forename: Jan
authors.initials: J
authors.lastname: Blatný
authors.forename: Prasad
authors.initials: P
authors.lastname: Mathew
authors.forename: Paul
authors.initials: P
authors.lastname: Monagle
authors.forename: Petra
authors.initials: P
authors.lastname: Ovesna
authors.forename: Veronika
authors.initials: V
authors.lastname: Fiamoli
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D015942:Factor VIIa / Q000009:adverse effects* / Q000627:therapeutic use*; D005260:Female; D006470:Hemorrhage / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D008297:Male; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use; D012042:Registries; D012189:Retrospective Studies
chemical_list: D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa