去纤苷在治疗肝小静脉闭塞病患者这方面效果如何？

Defibrotide, also known as defibrotide sodium, is currently recognized as the most promising new drug for the treatment of hepatic veno-occlusive disease (HVOD) after hematopoietic stem cell transplantation (HSCT). It was developed by Gentium Pharmaceuticals in Italy and later acquired by Jazz Pharmaceuticals in the United States. It was first launched in the EU in 2013 and was approved.

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 (sodium defibrotide) can act on these receptors to produce a variety of downstream effects. Its effect can also reduce the inflammatory damage of endothelial cells.

How effective is defibrotide in treating patients with hepatic veno-occlusive disease?

Defibrinoside increases tissue plasminogen activator (t-PA) and thrombomodulin expression, and decreases von Willebrand factor (vWF) and plasminogen activator inhibitor-1 (PAI-1) expression, thereby reducing EC activation and increasing EC-mediated fibrinolysis. Defibrotide protects ECs from chemotherapy, tumor necrosis factor-α (TNF-α), serum starvation, and perfusion-induced injury.

The effectiveness of defibrotide (defibrotide sodium) in the treatment of HVOD patients was explored in one study, which enrolled 19 patients with sHVOD. Give defibrotide treatment, intravenous infusion, 5~60mg/kg per day, the median use time is 15d (2-61d). The results showed that 42% of patients achieved complete remission (CR), and the overall survival rate 100 days after transplantation (OS) reached 32%. Patients also tolerated defibrotide relatively well. Since then, multiple clinical trials have confirmed the effectiveness of defibrotide in the treatment of HVOD.

To sum up, defibrotide (sodium defibrotide) can effectively alleviate the progression of the disease and prolong the overall survival rate of patients in the treatment of hepatic veno-occlusive disease, bringing new options to the treatment of patients.