去纤维钠治疗肝小静脉闭塞病患者效果如何？

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%. (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 defibrination is a safe and effective drug for preventing and treating HVOD after HSCT.

Based on the anticoagulant, fibrinolysis-promoting, and anti-inflammatory effects of defibrinated sodium, Richardson et al. first explored its application in the treatment of HVOD after HSCT. This study included 19 patients with sHVOD. The recommended dose of defibrotide sodium (defibrinoside) is 5~60 mg/kg per day, intravenous infusion, and the median usage time is 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%, and patients could tolerate defibrination well. Since then, many clinical trials have confirmed the effectiveness of defibrinated sodium in the treatment of HVOD.

In clinical trials of pediatric patients, defibrotide sodium also played a positive role, and the survival rate of patients was significantly improved. Moreover, studies have shown that the addition of other drugs for the treatment of HVOD does not affect the efficacy of defibrotide sodium in the treatment of HVOD.

Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said: "The approval of Defitelio fills a significant need for patients with rare but often fatal complications following chemotherapy and HSCT."

In summary, (Defibroside) has good effects in treating patients with hepatic veno-occlusive disease, both in adults and children, and is a good choice for patients.

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