Use of Off-label Recombinant Activated Factor Seven for Uncontrolled Bleeding

Clinical Center

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Abstract

This protocol is a chart review of the NIH Clinical Center's off-label use of rFVIIa for uncontrolled bleeding. rFVIIa is a prohemostatic agent that was first approved by the FDA in 1999 for use in patients with hemophilia A or B that have factors VIII or IX inhibitors. Since its introduction onto the market, other uses for rFVIIa have been explored, including use in patients with other coagulopathies or patients with normal coagulation that have uncontrolled or life-threatening bleeding, such as in trauma or intracerebral hemorrhage. Currently, off-label use of rFVIIa accounts for the majority of its use in the United States. Recently, several studies and meta-analyses of rFVIIa therapy for unapproved conditions have demonstrated minimal efficacy and possible harm from thrombotic complications in certain patient populations. At the NIH, we have a unique experience with the use of rFVIIa and have frequently used this medication off-label. Many of our patients are hematopoietic stem cell transplant recipients or have underlying disorders of hematopoiesis that impair their ability to achieve adequate hemostasis or make them prone to episodes of uncontrolled bleeding. Only limited data exists evaluating the use of rFVIIa in hematopoietic stem cell transplant recipients. The study will be limited to a review of patient medical records. This chart review began in the fall of 2011 and be completed by August 2013. We have identified all patients who have received rVIIa at the Clinical Center from 2003 2010. We have completed more than 50% of the data collection for these patients. This year we will extend our search to subjects that have received rFVIIa from 2010 - 2012 at the NIH. We will also add hematopoietic stem cell transplant subjects who developed DAH who did not receive rFVIIa, which will allow us to compare clinical outcomes in patients who did not receive rFVIIa versus those that did receive rFVIIa.