去纤维钠治疗肝小静脉闭塞病效果如何？

Also called defibrotide, it was approved for marketing in the EU in October 2013. It is currently recognized as the most promising new drug for the treatment of hepatic veno-occlusive disease (HVOD) after hematopoietic stem cell transplantation (HSCT). Defibrotide sodium was first developed by Italy's Gentium Pharmaceuticals and was approved by the U.S. Food and Drug Administration (FDA) for marketing in the United States on March 30, 2016.

Defibrotide sodium (defibrotide) is an injection that is not currently available in my country. Defibrinated sodium has the effects of anticoagulation, promoting fibrinolysis, and promoting blood vessel formation, and has played a significant role in preventing and treating HVOD.

Since many domestic patients do not know about defibrinated sodium, let’s take a closer look at its effectiveness in treating hepatic veno-occlusive disease.

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). Defibrotide sodium 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 can act on these receptors to produce a variety of downstream effects.

Let’s take a look at the performance of defibrinated sodium in clinical trials.

Usually, heparin can be used to prevent diseases such as hepatic veno-occlusion, but at the same time, the patient's risk of bleeding is also greatly increased. However, (defibrinoside) used alone or in combination with heparin has a very low incidence of side effects, and has achieved good results in preventing HV0D. In a phase III multicenter randomized clinical trial in Europe that included 356 stem cell transplant patients, the incidence of hepatic vein occlusion 30 days after transplantation was compared. The incidence of hepatic vein occlusion in the defibrotide group was 12% vs. 20% in the control group. Among them, only 2 of 180 patients in the defibrotide group developed hepatic vein occlusion; 35 of 176 patients in the control group developed hepatic vein occlusion. The results showed that defibrotide sodium has a significant effect in treating hepatic veno-occlusive disease.