Use of recombinant factor VIIa in treatment of bleeding episodes for patients with refractoriness to platelet transfusion therapy

Abstract

Abstract 4382Recombinant factor VIIa (rVIIa) is currently used for treatment and prevention of bleeding episodes in hemophilia with inhibitors, congenital factor VII deficiency, and acquired hemophilia. We report a series of 3 cases of immune mediated refractoriness to platelet transfusion therapy that were successfully treated with rVIIa, an off-label use. Data was obtained retrospectively over a 6 month period. All patients were known to be platelet refractory with presence of significant titers of plasma HLA antibodies. Two patients had undergone matched unrelated donor HSCT and the third patient suffered from Myelodysplastic syndrome (MDS). Two of the patients presented with GI bleeding and the third with intracranial bleeding. Due to limited availability of HLA matched platelets and negligible increment in platelet count from ABO matched platelets, use of factor VIIa was considered. Transfusion with PRBCs to maintain a Hemoglobin (Hb) > 8.0g/dl and close monitoring of coagulation parameters were performed. Patients received boluses of rFVIIa at 90mcg/kg, every 3–12 hrs. The dose of rFVIIa ranged from 10800–28,000 mcg/day. All three episodes of bleeding achieved clinical or radiologic improvement. No toxicities or arteriothrombotic events were observed with rFVIIa use. Our experience indicates that rFVIIa might be an effective treatment option for patients with refractoriness to platelet transfusion therapy. The optimal dosing schedule in this clinical situation needs to be studied.Patient demographics and outcomesPatientDiagnosisPre-intervention Hb/plateletrFVIIa used (microgms)PRBC/Platelet transfused (units)Clinical outcomeSV – GI bleedALL-MUD, BMT, day +102.7.9/400028000/d × 4 days6/3Bleeding stopped and Hemoglobin stabilized on day 4CS- GI bleedAML-MUD,BMT, day + 787.8/300025200/d × 2 days2/2Bleeding stopped and Hemoglobin stabilized on day 2MW-Intracranial hemorrhageMDS – IPSS risk group Intemediate-27.6/600010800/d × 2 days2/2Improvement in Neurological exam and stabilization on CT scan seen by day 3 Disclosures:Off Label Use: Recombinant factor VIIa for refractoriness to platelet transfusion therapy.