Objective: To determine the safety and efficacy of off label usage of Recombinant activated factor VII (rFVIIa) in children with severe bleeding in non-hemophiliac children with diverse etiologies like leukemia, post hematopoietic stem cell transplantation, dengue shock syndrome and Glanzmann thrombasthenia.
Method: Medical records of 16 non-hemophiliac children with 20 bleeding episodes where rFVIIa was administered only after failure of standard measures to control bleeding were retrospectively reviewed and data collected regarding patient demographics, diagnosis and location of bleeding. Blood counts, coagulation and other lab parameters, both pre and post rFVIIa, were also noted. Severity of bleeding was assessed using a scoring system used previously by Nevo S et al. A record of usage of the amount of blood components (red blood cells, platelets, fresh frozen plasma, and cryoprecipitate) 24 h pre and post rFVIIa was also made. The dose of rFVIIa and any adverse side effects were recorded.
Results: Bleeding sites were gastrointestinal (13/20), pulmonary hemorrhage (6/20) and intracranial hemorrhage (1/20). Thrombocytopenia (platelet count <50,000/cumm) was present in 50% episodes and five had refractory low platelets. Usage of packed red cells and platelets was significantly less after usage of rFVIIa (p value 0.001 and 0.006, respectively). Mean dosage of rFVIIa was 77 μgm/kg/dose (range 60 to 90 μgm/kg/dose). The bleeding stopped completely in 11(55%), decreased to minimal in 2 (10%), reduced but transfusions needed in 4 (20%) and did not stop in 3 (15%) episodes. Five patients (31%) did not survive the bleeding episode. None of the patients developed thrombosis.
Conclusions: rFVIIa appears to be safe and effective in controlling life-threatening bleed in non-hemophiliac children.