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Blood, 15 December 2004, Vol. 104, No. 13, pp. 3858-3864.
Prepublished online as a Blood First Edition Paper on August 24, 2004; DOI 10.1182/blood-2004-06-2223.
Previous Article | Next Article 
Submitted June 14, 2004
Accepted August 10, 2004
The use of recombinant factor VIIa in the treatment of bleeding disorders
Harold R Roberts*, Dougald M Monroe, and Gilbert C White
Division of Hematology/Oncology, Department of Medicine, University of North Carolina School of Medicine, Chapel Hill, NC, USA; Carolina Cardiovascular Biology Center, University of North Carolina School of Medicine, Chapel Hill, NC, USA
* Corresponding author; email: hrr{at}med.unc.edu.
Recombinant factor VIIa was initially developed for the treatment of hemorrhagic episodes in hemophilic patients with inhibitors to Factors VIII and IX respectively. After its introduction, it has also been used "off-label" to enhance hemostasis in non-hemophilic patients who experience bleeding episodes not responsive to conventional therapy. Evidence so far indicates that the use of factor VIIa in hemophilic patients with inhibitors is both safe and effective. Anecdotal reports also suggest that the product is safe and effective in controlling bleeding in non-hemophilic patients, However, its use in these conditions has not been approved by the FDA and conclusive evidence of its effectiveness from controlled clinical trials is not yet available. Several questions pertaining to the use of factor VIIa require further investigation including: the mechanism of action; the optimal dose; definitive indications; ultimate safety; and laboratory tests for monitoring therapy.

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