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Overview of the use of recombinant factor VIIa in children

Authors Puetz J

Received 10 July 2014

Accepted for publication 14 August 2014

Published 13 October 2014 Volume 2014:5 Pages 141—148

DOI https://doi.org/10.2147/PHMT.S50654

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 5

Editor who approved publication: Professor Laurens Holmes, Jr


John Puetz

Department of Pediatrics, Division of Hematology/Oncology, Cardinal Glennon Children's Medical Center, Saint Louis University, St Louis, MO, USA

Abstract: Recombinant factor VIIa (rFVIIa) was initially developed as a bypassing agent to control bleeding in people with hemophilia who have inhibitors. Due to its potential as a universal hemostatic agent, rFVIIa has also been used for a variety of other clinical indications. Investigations into the use of rFVIIa in children have demonstrated clinical efficacy and excellent safety when used for children with hemophilia and inhibitors. Investigations into the use of rFVIIa in children for alternative indications have been hampered by the lack of high-quality evidence. Most studies have shown that rFVIIa can be effective in reducing bleeding and blood product usage when given off-label in children. However, thrombotic events have been reported. In addition, no mortality benefit has been shown for rFVIIa. Despite this, over the last decade, the use of rFVIIa for indications beyond hemophilia has increased dramatically.

Keywords: rFVIIa, children, hemophilia, off-label, inhibitor, neonate

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