Defibrotide for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome with multiorgan failure. (November 2017)

• Record Type: Journal Article
• Authors: Richardson, Paul G; Grupp, Stephan A; Pagliuca, Antonio; Krishnan, Amrita; Ho, Vincent T; Corbacioglu, Selim
• Is Part Of: International journal of hematologic oncology. Volume 6:Number 3(2017)
• Page Start: 75
• Page End: 93
• Publication Date: 2017-11
• Subjects: defibrotide -- diagnostic criteria -- efficacy -- mechanism of action -- multiorgan dysfunction -- pathophysiology -- safety -- sinusoidal obstruction syndrome -- treatment -- veno-occlusive disease; Hematological oncology -- Periodicals; 616.99418
• DOI: 10.2217/ijh-2017-0015
• Languages: English
• ISSNs: 2045-1393

Abstract

Hepatic veno-occlusive disease, also called sinusoidal obstruction syndrome (VOD/SOS), is a potentially life-threatening and unpredictable complication of hematopoietic stem cell transplantation (HSCT). Characterized by a prothrombotic-hypofibrinolytic state, VOD/SOS typically presents with hyperbilirubinemia, ascites, weight gain and painful hepatomegaly; VOD/SOS with multiorgan failure may be associated with >80% mortality. Treatment has been mainly supportive. However, defibrotide is now approved in the USA for treatment of hepatic VOD/SOS with renal or pulmonary dysfunction following HSCT and in the European Union for treatment of severe hepatic VOD/SOS post-HSCT. In vitro evidence suggests defibrotide may restore thrombotic-fibrinolytic balance at the endothelial level and protect endothelial cells. Defibrotide has demonstrated significant reduction in VOD/SOS-related mortality and resolved VOD/SOS-related symptoms, with a manageable safety profile.