title: Safety and efficacy of defibrotide for the treatment of severe hepatic veno-occlusive disease.
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.
authors.affiliation: Dana-Farber Cancer Institute, 450 Brookline Avenue, Boston, MA 02115, USA.
authors.forename: Paul G
authors.initials: PG
authors.lastname: Richardson
authors.forename: Vincent T
authors.initials: VT
authors.lastname: Ho
authors.forename: Sergio
authors.initials: S
authors.lastname: Giralt
authors.forename: Sally
authors.initials: S
authors.lastname: Arai
authors.forename: Shin
authors.initials: S
authors.lastname: Mineishi
authors.forename: Corey
authors.initials: C
authors.lastname: Cutler
authors.forename: Joseph H
authors.initials: JH
authors.lastname: Antin
authors.forename: Nicole
authors.initials: N
authors.lastname: Stavitzski
authors.forename: Dietger
authors.initials: D
authors.lastname: Niederwieser
authors.forename: Ernst
authors.initials: E
authors.lastname: Holler
authors.forename: Enric
authors.initials: E
authors.lastname: Carreras
authors.forename: Robert
authors.initials: R
authors.lastname: Soiffer
keywords: defibrotide; sinusoidal obstruction syndrome; veno-occlusive disease