How effective is Defibrotide in treating hepatic veno-occlusive disease?

How effective is Defibrotide in treating hepatic veno-occlusive disease? Defibrotide was first developed by Gentium Pharmaceuticals of Italy. It was approved for marketing in the European Union in October 2013. It was approved for marketing in the United States by the U.S. Food and Drug Administration (FDA) on March 30, 2016, under the trade name Defitelio. Defibrotide is the first drug approved by the U.S. FDA to treat severe hepatic veno-occlusive disease. It is used for adult or pediatric patients with hepatic venous obstruction (VOD) and renal or pulmonary function abnormalities after receiving blood or bone marrow hematopoietic stem cell transplantation (HSCT).

Defibrotide is an adenosine receptor agonist with multiple effects. The adenosine A1/A2 receptors of endothelial cells are involved in the regulation of endothelial cells and the response of endothelial cells to injury. Defibrotide can act on these receptors to produce a variety of downstream effects. Defibrotide can also reduce the expression of cell adhesion molecules on the surface of endothelial cells, thereby reducing the adhesion of leukocytes to vascular endothelial cells and reducing the inflammatory damage of endothelial cells.

For some patients with leukemia and lymphoma, hematopoietic stem cell transplantation (HSCT) is the only hope to cure the disease and regain health. Hepatic veno-occlusive disease (HVOD) is one of the serious complications after hematopoietic stem cell transplantation (HSCT), and the mortality rate in patients with severe HVOD can be as high as 100%. Defibrotide (produced in Italy) is a single-stranded oligonucleotide mixture that has anti-thrombotic and fibrinolytic effects. In recent years, multiple clinical research results have shown that defibrotide is a safe and effective drug for preventing and treating HVOD.

  Richardson et al first explored the application of defibrotide in the treatment of HVOD after HSCT. This study included 19 patients with sHVOD. The dosage of defibrotide is 5~60mg/kg per day, intravenous infusion, and the median usage time is 15d (2-61d). The results showed that 42% of patients achieved complete remission (CR), and the overall survival (os) rate 100 days after transplantation reached 32%. The above is the question of the therapeutic effect of Defibrotide. If the patient has any other questions, please consult a medical partner.