The Effectiveness of Recombinant Factor VIIa in the Treatment of Bleeding in Pediatric Patients: A Ten-Year Experience from 1997 to 2007.

Introduction: Recombinant factor VIIa (rFVIIa) has been used in the treatment of bleeding in Thailand since 1997.

Methods: 125 patients receiving rFVIIa at the Faculty of Medicine, Ramathibodi Hospital and Regional Hospitals from 1997 to 2007 were retrospectively evaluated. Their age group ranged from newborn (n=11), infants (n=21), children (n=86) to adolescents (n=7). They were divided into 2 groups. Group 1 included 11 hemophiliacs with high inhibitor (A10, B1) exhibiting 90 episodes of bleeding or undergoing surgeries. The rFVIIa 100–200 μg/kg at 1–2 h interval for 1–4 doses combined with prednisolone, transamine, fibrin glue and dental splint were given. In cases of surgery, 100 μg/kg at 1–2 h interval was given in the first 24 h and extended to 3–4 h until the removal of stitches or 7–14 days after the surgical procedures. Sequential use of rFVIIa with non-activated or activated prothrombin complex concentrates were given to 9 patients. Group 2 included 114 non-hemophiliacs with or without pre-existing hemostatic disorder for controlling excessive bleeding unresponsive to conventional blood components or for preventing bleeding in invasive procedures. There were 122 episodes involving patients with Dengue Hemorrhagic Fever (n=38), massive bleeding (n=49), invasive procedure (n=23), Glanzmann thrombasthenia (n=6), congenital factor VII deficiency (n=5) and acquired aplastic anemia (n=1). For controlling excessive bleeding, 40–100 μg/kg of rFVIIa was given at 15–30 min interval for 1–4 doses until the bleeding significantly reduced, followed by 40–100 μg/kg at 2–4 h interval for 24–48 h until the bleeding was completely ceased. For preventing bleeding in invasive procedures, the similar regimen but with a lower fixed dose of 40–100 μg/kg was used. One to two doses were usually used. An exceptional high dose of 200–300 μg/kg was used in patients with Glanzmann thrombasthenia and a lower dose of 20–30 μg/kg at 6 h interval was used in patients with congenital factor VII deficiency.

Results: The efficacy of controlling bleeding in hemophiliacs revealed effective (80/90, 88.9%), partially-effective (6/90, 6.7%) and ineffective (4/90, 4.4%) responses while the non-hemophiliacs revealed effective (81/122, 88.5%), partially-effective (14/122, 11.5%) and ineffective (27/122, 22.1%) responses. The case-fatality rates were 0% and 27.2% (31/114) in hemophiliacs and non-hemophiliacs, respectively. Three episodes of thrombosis (3/125, 2.4%) were found 3 children underwent cardiac surgery.

Conclusion: rFVIIa has shown an effectiveness in the treatment of bleeding in pediatric patients with hemophiliacs and non-hemophiliacs.