Ligand id: 9087

Name: defibrotide

No information available.
Summary of Clinical Use
Approved to treat hepatic veno-occlusive disease (VOD) in adults and children following hematopoietic stem cell transplantation (HSCT)- reviewed in [2]. Defibrotide is the first and only approved treatment in Europe for severe VOD.
Phase III clinical trial results are published in [6]. A Phase II trial has been designed to evaluate defibrotide's anticoagulant effect in preventing sinusoidal obstruction syndrome in high-risk sickle cell patients undergoing a familial haploidentical allogeneic stem cell transplantation (NCT02675959) [1].
Mechanism Of Action and Pharmacodynamic Effects
Hepatic VOD can occur in patients who receive chemotherapy and HSCT, and it can lead to multiple organ failure and death. Although hepatic VOD is rare (~2% of patients develop the condition after HSCT), around 80% of these patient do not survive. Defibrotide, therefore fills a significant medical need in post-HSCT management.
Defibrotide's mechanism of action includes increasing PGI2 generation [3], with PGI2 reducing platelet aggregation. Defibrotide is also reported to have profibrinolytic activity in animal studies [4].
Hepatic VOD can also be known as sinusoidal obstruction syndrome (SOS).