Overview of the use of recombinant factor VIIa in children

John Puetz Department of Pediatrics, Division of Hematology/Oncology, Cardinal Glennon Children's Medical Center, Saint Louis University, St Louis, MO, USA Abstract: Recombinant factor VIIa (rFVIIa) was initially developed as a bypassing agent to control bleeding in people with hemophilia who have inhibitors. Due to its potential as a universal hemostatic agent, rFVIIa has also been used for a variety of other clinical indications. Investigations into the use of rFVIIa in children have demonstrated clinical efficacy and excellent safety when used for children with hemophilia and inhibitors. Investigations into the use of rFVIIa in children for alternative indications have been hampered by the lack of high-quality evidence. Most studies have shown that rFVIIa can be effective in reducing bleeding and blood product usage when given off-label in children. However, thrombotic events have been reported. In addition, no mortality benefit has been shown for rFVIIa. Despite this, over the last decade, the use of rFVIIa for indications beyond hemophilia has increased dramatically. Keywords: rFVIIa, children, hemophilia, off-label, inhibitor, neonat