Veno-occlusive Hepatic Disease (Sinusoidal Obstruction Syndrome) Medication

Updated: Apr 24, 2021
  • Author: James L Harper, MD; Chief Editor: Jennifer Reikes Willert, MD  more...
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Medication Summary

In the United States, defibrotide is the only FDA-approved drug showing significant results for treating veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) compared with historical controls. The mechanism of action has not been fully elucidated, but defibrotide is thought to enhance the enzymatic activity of plasmin to hydrolyze fibrin clots. It elicits antithrombotic and fibrinolytic properties and may increase prostaglandin E2 and prostacyclin levels, alter platelet activity, and increase tissue plasminogen activator function while decreasing tissue plasminogen activator inhibitor activity.



Antifibrinolytic/Antithrombotic Agent

Class Summary

Defibrotide is a single-stranded polydeoxyribonucleotide derived from porcine tissue that possesses antithrombotic, thrombolytic, anti-inflammatory, and anti-ischemic properties.

Defibrotide (Defitelio)

The mechanism of action has not been fully elucidated. It enhances the enzymatic activity of plasmin to hydrolyze fibrin clots. Studies evaluating the pharmacological effects of defibrotide on endothelial cells (ECs) were conducted primarily in the human microvascular endothelial cell line. In vitro, defibrotide increased tissue plasminogen activator (t-PA) and thrombomodulin expression and decreased von Willebrand factor (vWF) and plasminogen activator inhibitor-1 (PAI-1) expression, thereby reducing EC activation and increasing EC-mediated fibrinolysis. It protects ECs from damage caused by chemotherapy, TNF-alpha, serum starvation, and perfusion.

It is indicated for adults and children with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT).