Evaluating the Effectiveness of Recombinant Factor VIIa in Nonhemophilic Pediatric Cancer Patients with Acute Severe Bleeding

Abstract

Abstract Context: Despite recombinant factor VIIa (rFVIIa) being approved for treating bleeding in hemophilia patients, there has been limited research on its efficacy in nonhemophilic cases, particularly, in pediatric cancer patients who are vulnerable to bleeding. Aim: Our objective is to assess rFVIIa’s effectiveness in managing severe bleeding in pediatric nonhemophilic cancer patients. Settings and Design: We conducted a retrospective study at a comprehensive cancer center. Materials and Methods: It involves reviewing cases of nonhemophilic pediatric cancer patients treated with rFVIIa for acute severe bleeding during hospitalization between 2013 and 2022 using the pharmacy electronic system. Patients without a cancer diagnosis were excluded. The primary outcome assessed was the cessation of bleeding within 48 h post the last administered rFVIIa dose. Statistical Analysis Used: Descriptive statistics were used to report the outcomes. Results: The results revealed that our analysis encompassed 22 nonhemophilic pediatric cancer patients experiencing acute severe bleeding. The mean age was 7 ± 16.9 (SD) years, with 18 (81.8%) being male. Among them, 7 (31.8%) had leukemia and 5 (22.7%) had neuroblastoma. Thrombocytopenia was prevalent in 21 (95.5%) patients, and 7 (31.8%) underwent bone marrow transplantation. On average, patients received 85 ± 10 (SD) µg/kg of rFVIIa for one to two doses per bleeding episode. Bleeding cessation was achieved in 13 (59%) patients. Conclusion: Our findings suggest that rFVIIa could serve as a viable therapeutic option for managing acute severe bleeding in nonhemophilic pediatric cancer patients.