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

Research output: Contribution to journalArticle

29 Citations (Scopus)

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

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Hepatic Veno-Occlusive Disease
Stem Cell Transplantation
Safety
Hematopoietic Stem Cell Transplantation
Oligonucleotides
Survival
Mortality
defibrotide
Therapeutics

All Science Journal Classification (ASJC) codes

  • Hematology

Cite this

Richardson, P. G., Vincent, T., Cutler, C., Antin, J. H., Stavitzski, N., Soiffer, R., ... 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
Richardson, Paul G. ; Vincent, T. ; Cutler, Corey ; Antin, Joseph H. ; Stavitzski, Nicole ; Soiffer, Robert ; Giralt, Sergio ; Arai, Sally ; Mineishi, Shin ; Niederwieser, Dietger ; Holler, Ernst ; Carreras, Enric. / Safety and efficacy of defibrotide for the treatment of severe hepatic veno-occlusive disease. In: Therapeutic Advances in Hematology. 2012 ; Vol. 3, No. 4. pp. 253-265.
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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.",
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Richardson, PG, Vincent, T, Cutler, C, Antin, JH, 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, vol. 3, no. 4, pp. 253-265. https://doi.org/10.1177/2040620712441943

Safety and efficacy of defibrotide for the treatment of severe hepatic veno-occlusive disease. / Richardson, Paul G.; Vincent, T.; Cutler, Corey; Antin, Joseph H.; Stavitzski, Nicole; Soiffer, Robert; Giralt, Sergio; Arai, Sally; Mineishi, Shin; Niederwieser, Dietger; Holler, Ernst; Carreras, Enric.

In: Therapeutic Advances in Hematology, Vol. 3, No. 4, 08.2012, p. 253-265.

Research output: Contribution to journalArticle

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AU - Richardson, Paul G.

AU - Vincent, T.

AU - Cutler, Corey

AU - Antin, Joseph H.

AU - Stavitzski, Nicole

AU - Soiffer, Robert

AU - Giralt, Sergio

AU - Arai, Sally

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