去纤维钠对肝小静脉闭塞病有多大的疗效？

(Defibrotide) can be used to treat hepatic veno-occlusive disease. The recommended dose is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. In recent years, multiple clinical research results have shown that defibrotide sodium is a safe and effective drug for preventing and treating HVOD after HSCT.

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

In vitro, defibrinated sodium enhances the enzymatic activity of plasmin that hydrolyzes fibrin clots, increases tissue plasminogen activator (t-PA) and thrombomodulin expression, and decreases von Willebrand factor (vWF) and plasminogen activation inhibitor-1 (PAI-1) expression, thereby reducing EC activation and increasing EC-mediated fibrinolysis. Defibrinated sodium protects ECs from chemotherapy, tumor necrosis factor-alpha (TNF-alpha), serum starvation, and perfusion-induced injury.

In clinical trials, heparin was used to prevent diseases such as hepatic venous occlusion while greatly increasing the risk of bleeding. However, (defibrinoside) used alone or in combination with heparin had a very low incidence of side effects and achieved good results in preventing HVOD. In a phase III multi-center randomized clinical trial completed in Europe, a total of 356 stem cell transplant patients were recruited. The incidence of hepatic vein occlusion 30 days after transplantation was compared. It was found that 2 of 180 patients in the defibrinated sodium group developed hepatic vein occlusion, accounting for 12%; 35 of 176 patients in the control group developed hepatic vein occlusion, accounting for 20%.