去纤苷对肝小静脉闭塞病能有多大疗效？

Defibrotide (sodium defibrotide, produced in Italy) is a mixture of single-stranded oligonucleotides with antithrombotic and fibrinolytic effects. In recent years, multiple clinical research results have shown that defibrotide is a safe and effective drug for preventing and treating hepatic veno-occlusive disease HVOD.

Defibrotide (sodium 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 (defibrinated sodium) 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, defibrotide (defibrotide sodium) 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 defibrotide can significantly increase the expression of thrombin regulatory protein (TM) and tissue factor pathway inhibitors to produce anticoagulant effects.

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%. The launch of defibrotide has brought new options for the treatment of 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%. Defibrotide is effective in treating hepatic veno-occlusive disease.