defiteli治疗肝小静脉闭塞病效果如何？

(Defibrotide) is a single-stranded oligonucleotide mixture with anti-thrombotic and fibrinolysis-promoting effects. In recent years, multiple clinical study results have shown that defiteli is a safe and effective drug for the prevention and treatment of hepatic veno-occlusive disease after HSCT.

On March 30, 2016, the FDA approved defibrotide (defiteli) for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), and renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT).

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

Defiteli 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. Defiteli 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. In addition, defiteli can also promote the release of prostaglandin I2 (PGI2) and prostaglandin E2 (PGE2), thereby causing blood vessel dilation, inhibiting platelet aggregation, and reducing ischemic damage. Studies have shown that defiteli can significantly increase the expression of thrombin regulatory protein (TM) and tissue factor pathway inhibitors to produce anticoagulant effects.

In one study, 19 patients with sHVOD were included. (Defibrinoside) dosage 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%, and patients could tolerate defiteli well. Since then, multiple clinical trials have confirmed the effectiveness of defiteli in the treatment of hepatic veno-occlusive disease.