去纤苷治疗肝小静脉闭塞病的效果好吗？

Is defibrotide (defibrotide sodium) effective in treating hepatic veno-occlusive disease? Let's take a look. Defibrotide is indicated for the treatment of adults and children with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), and renal or pulmonary dysfunction after hematopoietic stem cell transplantation (HSCT). It was approved for marketing in the EU in 2013 and in the United States in 2016. The drug was also granted "orphan drug" status by the FDA.

Some complications may occur after hematopoietic stem cell transplantation (HSCT), and hepatic veno-occlusive disease (VOD) is one of its serious complications. The mortality rate in patients with severe VOD can be as high as 100%. Defibrotide (sodium defibrotide) is a mixture of single-stranded oligonucleotides with anti-thrombotic and fibrinolysis-promoting effects. In recent years, multiple clinical research results have shown that defibrotide is a safe and effective drug for preventing and treating VOD after HSCT.

In a pilot study, 528 patients diagnosed with VOD symptoms after HSCT were included in 3 studies to evaluate the effectiveness of defibrotide (defibrotide sodium). These patients also had abnormal liver or renal function. The evaluation index is the overall survival rate 100 days after HSCT. The results showed that the survival rate of patients who received defibrotide treatment after 100 days was 38% to 45%; while data analysis found that the survival rate of HSCT patients who were expected to receive only supportive care or other drug intervention after 100 days was only 21% to 31%. The use of defibrotide can effectively improve the survival rate of patients. In clinical trials, heparin is usually used to prevent diseases such as hepatic veno-occlusion, which also greatly increases the patient's risk of bleeding. However, defibrotide alone or in combination with heparin has a low incidence of side effects, good safety, and has achieved good VOD prevention effects. Defibrinoside (Defibrotide Sodium) is also currently recognized as a relatively effective drug in the treatment of hepatic veno-occlusive disease (VOD).