去纤苷对肝小静脉闭塞病有效果吗？

Is defibrotide (sodium defibrotide) effective in treating hepatic veno-occlusive disease? Defibrotide (defibrotide sodium) is currently recognized as the most promising new drug for the treatment of hepatic veno-occlusive disease (HVOD) after hematopoietic stem cell transplantation (HSCT). Based on its anticoagulant, fibrinolysis-promoting, and anti-inflammatory effects, Richardson et al. first explored its application in the treatment of HVOD after HSCT. This study included 19 sHVOD patients. The dosage of defibrotide 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 defibrination well. Since then, multiple clinical trials have confirmed the effectiveness of defibrotide in the treatment of HVOD. 

The recommended dose of defibrotide (Defibrotide Sodium) for adult and pediatric patients is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. The dose should be based on the patient's baseline weight, which is defined as the patient's weight before the HSCT preparation protocol. Defibrotide was administered for a minimum of 21 days. If signs and symptoms of hepatic VOD have not resolved after 21 days, continue defibrination until resolution of VOD or up to a maximum of 60 days. Defibrotide must be diluted prior to infusion.

Defibrotide (sodium defibrotide) is a mixture of single-stranded deoxyoligonucleotide sodium salts produced by controlled depolymerization of porcine intestinal mucosal genomic DNA. Its mechanism of action is complex and has not yet been fully elucidated. The effectiveness of defibrotide (defibrotide sodium) was evaluated through 3 research trials on 528 patients. The subjects participating in the 3 trials were all patients diagnosed with VOD symptoms after HSCT and accompanied by abnormal liver or renal function. The overall survival rate 100 days after HSCT was used as the evaluation Indicators and results showed that the survival rate after 100 days for patients treated with defibrotide (sodium defibrotide) was 38% to 45%; while data analysis found that the survival rate after 100 days for HSCT patients who were expected to receive only supportive care or other drug intervention was only 21% to 31%. Note that defibrotide should not be used in patients with bleeding complications and those taking blood thinners or other medications that reduce blood clotting. 

Defibrotide (sodium defibrotide) also has a certain therapeutic effect on multiple myeloma. It has achieved good results in the treatment of multiple myeloma and hepatic veno-occlusive disease caused by chemotherapy and stem cell transplantation, meeting the treatment needs of this rare and fatal disease.