Defibrotide sodium for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome.

Journal Article (Journal Article;Review)

Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is an unpredictable condition associated with endothelial-cell damage due to conditioning for hematopoietic stem-cell transplantation (HSCT) or chemotherapy without HSCT. Mortality in patients with VOD/SOS and multi-organ dysfunction (MOD) may be >80%. Areas covered: Defibrotide is the only approved drug for the treatment of severe hepatic VOD/SOS after HSCT in the European Union and hepatic VOD/SOS with renal or pulmonary dysfunction in the United States. Its efficacy in patients with VOD/SOS with MOD post-HSCT was demonstrated in a clinical-trial program that included a historically controlled treatment study, a phase 2 trial, and a large T-IND expanded-access program that also included patients without MOD and who received chemotherapy without HSCT. Expert commentary: Defibrotide appears to protect endothelial cells and restore the thrombolytic-fibrinolytic balance. It addresses a significant clinical need and has demonstrated favorable Day +100 survival and overall adverse-event rates that seem similar to control groups receiving supportive care alone. Currently, defibrotide is under investigation for the prevention of VOD/SOS in high-risk pediatric and adult patients.

Full Text

Duke Authors

Cited Authors

  • Richardson, PG; Triplett, BM; Ho, VT; Chao, N; Dignan, FL; Maglio, M; Mohty, M

Published Date

  • February 2018

Published In

Volume / Issue

  • 11 / 2

Start / End Page

  • 113 - 124

PubMed ID

  • 29301447

Electronic International Standard Serial Number (EISSN)

  • 1751-2441

Digital Object Identifier (DOI)

  • 10.1080/17512433.2018.1421943


  • eng

Conference Location

  • England