Clinical Outcomes of Off-Label Use of Recombinant Activated Factor VII (rFVIIa) at Teaching Hospitals Affiliated with a Medical School.

BACKGROUND: Recombinant factor VIIa (rFVIIa), NovoSeven^®, is currently licensed in the United States for treatment of bleeding in hemophilia patients with acquired coagulation factor inhibitors. The off-label uses of this medication have expanded dramatically in past few years. Here we report our experience with the off-label use of rFVIIa at University at Buffalo affiliated teaching hospitals which include a level III trauma and major cardiothoracic centers.

PATIENTS AND METHODS: We reviewed the clinical, laboratory, and radiographic data of a consecutive series of 43 patients who received off-label rFVIIa between December 2001 and September 2005.

RESULTS: The indications for off-label rFVIIa use included CNS bleeding (17 patients), cardiothoracic and vascular surgery (9 patients), trauma (7 patients), GI bleeding (4 patients), coagulopathy (2 patients) and prophylaxis for surgery (4 patients). The dosage ranged from 32 to 150 mcg/kg with most patients receiving 90 mcg/kg. While a majority of patients received a single dose of rFVIIa (29 patients), one received a total of 22 doses. Overall 21 patients (49%) were alive at discharge. Those patients who received rFVIIa to stop severe bleeding from trauma or surgery appeared to have the worst outcome. The patient who received 22 doses of rFVIIa appeared to derive some clinical benefit after his initial doses, but became non-responsive to additional therapies. Successful hemostasis with rFVIIa was achieved in 100% of patients with preoperative prophylactic use. There were no thromboembolic events attributed to rFVIIa treatment.

CONCLUSIONS: The outcome of off-label use of rFVIIa appears to correlate with clinical indications, with prophylactic use having greatest benefit and surgical hemorrhage, the worst. Our findings were used to revise guidelines to allow for more efficacious use of off-label rFVIIa.