Authors' objectives:

This report evaluates the level of evidence currently available to support the effectiveness and safety of using recombinant activated coagulation factor VII (rFVIIa) for clinical indications beyond those approved by the Food and Drug Administration (FDA). rFVIIa is approved for a variety of uses in hemophilia patients who have developed antibody inhibitors that compromise the use of standard factor replacement. Use of this costly biologic product has expanded beyond these hemophilia-related indications to encompass a range of off-label uses that differ from the drug’s FDA approved label. The purpose of this report is two-fold: (1) To profile the full range of clinical indications for which rFVIIa is being used and the types of studies available to evaluate these uses, and (2) To provide a comparative effectiveness review of rFVIIa versus usual care for several clinical indications: intracranial hemorrhage, massive bleeding secondary to trauma, and the selected surgical procedures of cardiac surgery, liver transplantation, and prostatectomy.

Authors' recommendations: Available evidence on off-label rFVIIa use is limited across a wide spectrum of off-label indications. Considering the evidence as a whole, off-label rFVIIa may provide some benefit for certain clinical indications, but this conclusion is largely based on indirect outcomes that have an uncertain relationship to patient survival or functional status. Of the indications we studied, the benefit-to-risk ratio may be more favorable for body trauma than for other indications, because its use may reduce the occurrence of acute respiratory distress syndrome (ARDS); however, the strength of evidence is low for this as well as most other outcomes, which precludes definitive conclusions. Available evidence does not indicate that use of off-label rFVIIa reduces mortality or improves other direct outcomes for the indications we studied. Thromboembolic events are increased by use of rFVIIa in intracranial hemorrhage and adult cardiac surgery. Despite this state of evidence, in-hospital, off-label cases of rFVIIa use have increased in the last decade, particularly for cardiac surgery, trauma, and intracranial hemorrhage.

Project Status: Completed

URL for project: http://www.effectivehealthcare.ahrq.gov/ehc/products/20/452/Off-Label%20exec%20summ.pdf

Year Published: 2010

URL for published report: http://www.effectivehealthcare.ahrq.gov/ehc/products/20/450/Final%20Report_CER21_Factor7.pdf

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: United States

Organisation Name: Agency for Healthcare Research and Quality

Contact Address: Center for Outcomes and Evidence Technology Assessment Program, 540 Gaither Road, Rockville, MD 20850, USA. Tel: +1 301 427 1610; Fax: +1 301 427 1639;

Contact Name: martin.erlichman@ahrq.hhs.gov

Contact Email: martin.erlichman@ahrq.hhs.gov

Copyright: Agency for Healthcare Research and Quality (AHRQ)