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Defibrotide in the treatment of hepatic veno-occlusive disease

Authors Fulgenzi A, Ferrero ME

Received 5 July 2016

Accepted for publication 8 September 2016

Published 31 October 2016 Volume 2016:8 Pages 105—113

DOI https://doi.org/10.2147/HMER.S79243

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Amy Norman

Peer reviewer comments 3

Editor who approved publication: Dr Gerry Lake-Bakaar


Alessandro Fulgenzi, Maria Elena Ferrero

Department of Biomedical Sciences for Health, University of Milan, Milan, Italy

Abstract: Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), represents the most frequent complication in patients in early phase following hematopoietic stem-cell transplantation (HSCT). In its severe form, VOD/SOS can be associated with multiorgan failure and with a mortality rate >80% by day +100. Defibrotide (DF) (a mixture of 90% single-stranded phosphodiester oligonucleotides and 10% double-stranded phosphodiester oligonucleotides derived from controlled depolarization of porcine intestinal mucosal DNA) has been proposed for the treatment of SOS due to its ability to restore thrombo-fibrinolytic balance and protect endothelial cells. The present review highlights why the mechanisms of action of DF allow its successful use in the prevention and treatment of SOS following HSCT.

Keywords: hepatic veno-occlusive disease, hematopoietic stem-cell transplantation, defibrotide

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