肝小静脉闭塞病患者用去纤维钠治疗的效果

It is a very effective drug for preventing and treating hepatic veno-occlusive disease (HVOD) after hematopoietic stem cell transplantation (HSCT). It is also called defibrotide. It was developed by Jazz Company and was approved for marketing in the EU in October 2013. HVOD is one of the serious complications of HSCT. The mortality rate of patients with severe HVOD can be as high as 100%, and the risk of death is extremely high. Defibrotide sodium is currently recognized as the most promising new drug for the treatment of HVOD.

Defibrotide sodium is used to treat rare diseases and has received widespread attention since its launch. Among them, what everyone wants to know most is the effect of its treatment on patients with hepatic veno-occlusive disease. Let’s learn about it from some trials.

The effectiveness of defibrotide sodium (defibrinoside) in the treatment of HVOD patients was explored in one study, which enrolled 19 patients with sHVOD. Give defibrinated sodium treatment, intravenous infusion, 5~60 mg/kg per day, the median use time is 15 days (2-61 days). The results showed that 42% of patients achieved complete remission (CR), and the overall survival rate 100 days after transplantation (OS) reached 32%. Patients also tolerated defibrinated sodium relatively well. In addition, many trials have confirmed that defibrotide (defibrotide) is safe and effective in the treatment of hepatic veno-occlusive disease (HVOD).

It has also been granted "orphan drug" status by the FDA, which can be used not only by adults but also by children. In order for defibrotide sodium to be effective in treating hepatic venous occlusion and venous occlusion, patients need to take the medication correctly. The recommended dose of defibrinated sodium is 6.25 mg/kg given as a 2-hour intravenous infusion every 6 hours based on body weight. The minimum duration of treatment with defibrotide is 21 days.

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