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. 2012 Aug;3(4):253-65.
doi: 10.1177/2040620712441943.

Safety and efficacy of defibrotide for the treatment of severe hepatic veno-occlusive disease

Paul G Richardson  1 , Vincent T Ho, Sergio Giralt, Sally Arai, Shin Mineishi, Corey Cutler, Joseph H Antin, Nicole Stavitzski, Dietger Niederwieser, Ernst Holler, Enric Carreras, Robert Soiffer
Affiliations

Safety and efficacy of defibrotide for the treatment of severe hepatic veno-occlusive disease

Paul G Richardson et al. Ther Adv Hematol. 2012 Aug.

Abstract

Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome, is a potentially life-threatening complication of chemotherapeutic conditioning used in preparation for hematopoietic stem-cell transplantation (SCT). VOD may occur in up to 62% of patients undergoing SCT, with onset generally within the first month after SCT. In severe cases, 100-day mortality is in excess of 80%. Current management consists of best supportive care, with no agents to date approved for treatment in the USA or the EU. Defibrotide, a polydisperse oligonucleotide, has been shown in phase II and III trials to improve complete response and survival in patients undergoing SCT with severe VOD. This article reviews our current understanding of VOD, and examines recent clinical findings on defibrotide for the treatment and prophylaxis of VOD.

Keywords: defibrotide; sinusoidal obstruction syndrome; veno-occlusive disease.

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Conflict of interest statement

Conflict of interest statement: V.T. Ho, S. Giralt, S. Arai, S. Mineishi, J.H. Antin and N. Stavitzski have no conflicts of interest to declare. P.G. Richardson is on the advisory board for Gentium S.p.A. C. Cutler is consultant for Sigma Tau and has equity in Gentium S.p.A. D. Niederwieser is a consultant for the speakers bureau, Gentium S.p.A. E. Holler has received honoraria and research grants from Gentium S.p.A. E. Carreras and R. Soiffer have received honoraria from Gentium S.p.A.

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