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

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

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32 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)253-265
Number of pages13
JournalTherapeutic Advances in Hematology
Volume3
Issue number4
DOIs
StatePublished - Aug 2012

All Science Journal Classification (ASJC) codes

  • Hematology

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    Richardson, P. G., Vincent, T., Cutler, C., Antin, J. H., Stavitzski, N., Soiffer, R., Giralt, S., Arai, S., Mineishi, S., Niederwieser, D., Holler, E., & Carreras, E. (2012). Safety and efficacy of defibrotide for the treatment of severe hepatic veno-occlusive disease. Therapeutic Advances in Hematology, 3(4), 253-265. https://doi.org/10.1177/2040620712441943