去纤苷治疗肝小静脉闭塞病的效果怎么样？

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%. Defibrinoside (Defibrotide Sodium) is a mixture of single-stranded oligonucleotides with antithrombotic and fibrinolytic effects. In recent years, multiple clinical research results have shown that defibrotide (defiteli) is a safe and effective drug for preventing and treating HVOD. 

In vitro, defibrotide (sodium defibrotide) 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 activator inhibitor-1 (PAI-1) expression, thereby reducing EC activation and increasing EC-mediated fibrinolysis. Defibrotide (defiteli) protects ECs from damage caused by chemotherapy, tumor necrosis factor-alpha (TNF-alpha), serum starvation, and perfusion.

In clinical trials, heparin was used to prevent diseases such as hepatic venous occlusion while greatly increasing the risk of bleeding. However, defibrotide (defibrotide sodium) alone or in combination with heparin has a very low incidence of side effects and has achieved a good effect in preventing HV0D. 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 defibrotide (sodium defibrotide) group developed hepatic vein occlusion, accounting for 12%; 35 of 176 patients in the control group developed hepatic vein occlusion, accounting for 20%.  

Defibrotide (Defibrotide Sodium) is the first drug approved by the FDA for the treatment of severe hepatic venular occlusion, a rare and fatal liver disease. The approval was based on a Phase 2 trial of 75 patients, a Phase 3 trial of 102 patients, and an expansion trial of an additional 351 patients. All patients in the trial were diagnosed with hepatic veno-occlusive disease and associated renal or pulmonary dysfunction after HSCT (hematopoietic stem cell transplantation). In the phase 2 trial, 44% of patients survived 100 days after transplantation, compared with 38% in the phase 3 trial and 45% in the expanded trial, and the FDA noted in a report that the expected 100-day survival rate for patients with severe veno-occlusive disease who were not treated with defibrotide was 21%-31%. 

The above is the effect of defibrotide (sodium defibrotide) in the treatment of hepatic veno-occlusive disease provided by our Medical Companions Overseas Medical Consulting Service Company. From this point of view, the effect of defibrotide (sodium defibrination) cannot be underestimated.