defiteli对肝小静脉闭塞病有效果吗？

On March 30, 2016, the FDA approved defibrotide(R) to treat adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), and renal or pulmonary dysfunction following hematopoietic stem cell transplantation (HSCT).

Studies have shown that defiteli exerts various biological functions mainly by affecting vascular endothelial cell response and platelet activity. Defibrinoside contains nucleic acid aptamers that can interact with adenosine A1 and A2 receptors, thereby reducing the response of endothelial cells to injury and protecting vascular endothelium. In vitro experiments and in vivo animal experiments have confirmed that defiteli can stimulate the release of prostacyclin PGI2. PGI2 is mainly synthesized and secreted by vascular endothelium and platelets. It dilates blood vessels and inhibits platelet aggregation by increasing the level of cAMP. The increase of PGI2 can also inhibit the lipoxygenase pathway to reduce the production of leukotriene LTB4 and reduce the damage of tissue from ischemia.

Hepatic veno-occlusive disease (VOD) is a common complication of high-dose chemotherapy and hematopoietic stem cell transplantation. It causes damage to liver sinusoidal endothelial cells and hepatocytes, accompanied by progressive venule occlusion, eventually leading to hepatocyte necrosis. VOD usually occurs around 30 days after transplantation, with an average incidence of 14%. Some patients have mild symptoms, while severe cases may develop multiple organ failure (MOF), and the 100-day survival rate is less than 15%.

In one study, 19 patients with sHVOD were included. The dosage is 5~60mg/kg per day, intravenous infusion, and the median usage time is 15d (2-61d). The results showed that 42% of patients achieved complete remission (CR), and the overall survival (os) rate 100 days after transplantation reached 32%, and patients could tolerate defiteli well. Since then, multiple clinical trials have confirmed the effectiveness of defiteli in the treatment of hepatic veno-occlusive disease.