Purpose: Unlabeled uses of factor VIIa (recombinant) in pediatric patients are reviewed.
Summary: Factor VIIa (recombinant) is currently approved for the treatment and prevention of bleeding in patients with hemophilia A or B and inhibitors of coagulation factors, acquired hemophilia, or congenital factor VII deficiency. Use of this agent has expanded to include unlabeled indications, including bleeding unrelated to coagulation factor deficiencies in infants, children, and adolescents without congenital hemophilia. Results of a search of the English-language medical literature for relevant articles primarily included case reports and retrospective reviews, with few randomized clinical trials. Reasons for use of factor VIIa (recombinant) included bleeding associated with acquired coagulopathies or congenital disorders resulting in coagulopathies, hepatic failure, surgery, and bleeding associated with prematurity, malignancies, and trauma. In most reports, conventional therapies were used with limited or no success. Factor VIIa (recombinant) was most commonly used in patients with coagulopathies or hemorrhage secondary to surgical procedures, primarily cardiopulmonary bypass and liver transplantation, as well as intracranial hemorrhage. In general, higher mortality rates were reported in medical versus surgical patients. The lowest rates of complete response were seen in younger patients and patients with trauma. A decrease in the requirement of blood product transfusion after the use of factor VIIa (recombinant) versus standard therapies or placebo was commonly observed but was not statistically significant in many cases.
Conclusion: Given the lack of well-designed controlled studies, current evidence is inconclusive regarding the safety and efficacy of factor VIIa (recombinant) for unlabeled indications in pediatric patients.