Defibrotide for Prevention and Treatment of Veno-Occlusive Disease in Adults

Hepatic veno-occlusive disease (VOD) -also known as sinusoidal obstruction syndrome- is considered one of the leading causes of morbidity and mortality after hematopoietic stem cell transplantation (HSCT). Pathophysiology is thought to be multifactorial: endothelial injury in both sinusoids and small hepatic venules leading to subendothelial deposition of platelet and coagulative material, which results in centrilobular necrosis and post-sinusoidal obstruction. Defibrotide is a mixture of porcine oligodeoxyribonucleotides that has antithrombotic, anti-ischemic and anti-inflammatory properties.

This agent seems to have a protective effect against endothelial cell injury by increasing fibrinolysis and reducing procoagulant activity yet without an increase in bleeding events. In support of these observations, several studies focused attention on the use of defibrotide as a potentially effective and reliable agent for preventing and treating VOD. Defibrotide use was first reported in patients diagnosed with severe VOD following stem cell transplantation performed between 1995 and 1997. Since then, several studies in both adults and children have demonstrated encouraging responses with defibrotide in severe VOD. Read more>>>>>>>>>>>>>>>