去纤维钠治疗效果怎么样？

(Defibrinoside, defiteli) is a single-stranded oligonucleotide mixture with anti-thrombotic and fibrinolytic effects. It was approved for marketing in the European Union in 2013 and by the US FDA in 2016. It is a DNA-derived anticoagulant used for the treatment of hepatic veno-occlusive disease and hematopoietic stem cell transplantation for renal or pulmonary insufficiency.

Leukemia and lymphoma are malignant tumors that threaten human life and health. For some patients, 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), and the mortality rate in patients with severe HVOD can be as high as 100%. The launch of defibrinated sodium has reduced the risk of HVOD to a great extent.

Defibrinated sodium has not yet been launched in our country. Let’s take a look at the treatment effect of defibrated sodium.

Richardson et al first explored its application in the treatment of HVOD after HSCT. In this trial, a total of 19 sHVOD patients participated in the treatment. Defibrotide sodium was administered at 5~60 mg/kg daily through intravenous infusion, and the median usage time was 15 days (2-61 days). The results showed that 42% of patients achieved complete remission (CR), and the overall survival (os) rate 100 days after transplantation reached 32%. The effect is very obvious.

In a phase 2 trial of 75 patients, 44% survived 100 days after transplantation, in a phase 3 trial of 102 patients, 38% survived 100 days, and in an expanded trial of an additional 351 patients, 45% survived 100 days, all of whom were diagnosed with hepatic VOD and associated renal or pulmonary dysfunction after HSCT. The estimated 100-day survival rate for patients with untreated severe hepatic VOD is 21%-31%. Therefore, the use of defibrinated sodium effectively prolongs the survival time of patients.