Single Higher Dose of Recombinant Activated Factor VII in the Treatment of Hemorrhages in Patients with Hemophilia Complicated by Inhibitors

The development of inhibitors is the most severe complication of treating hemophilia patients with Factor VIII or IX, and providing effective hemostasis for inhibitor patients is challenging. Patients with high responding inhibitors (titer >5 BU/mL) are usually treated with recombinant activated Factor VII (rFVIIa), 90-120 mu g/kg every 2-3 hours, or plasma-derived activated prothrombin complex concentrate. The aim of this study was to assess the efficacy and safety of a single dose of 270 mu g/kg rFVIIa in inhibitor patients with joint or soft tissue bleeds treated at the Department of Paediatric Hematology and Oncology and the Department of Hematology and Transplantology. 7 inhibitor patients (3 adults aged 23-33 and 4 children aged 3-14) were included in the study. The hemostatic efficacy and tolerability of a single high dose of rFVIIa for home treatment of moderate or severe bleeds was evaluated. Treatment with rFVIIa effectively stopped bleeding in all patients without any adverse events. In most cases a single dose of rFVIIa 270 mu g/kg was more effective than 3 or 4 lower doses (90 mu g/kg) and less traumatic for pediatric patients. Based on present studies, it can be concluded that a single dose of rFVIIa is effective and well tolerated in the treatment of intra-articular bleeds in patients with hemophilia complicated by the presence of inhibitors (Adv Clin Exp Med 2012, 21, 4, 519-524).