去纤苷治疗肝小静脉闭塞病效果怎样？

Defibrotide 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 sodium and defiteli. 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 is currently recognized as the most promising new drug for the treatment of HVOD.

How effective is defibrotide in treating hepatic veno-occlusive disease?

The effectiveness of defibrotide (defibrotide sodium) in the treatment of HVOD patients was explored in one study, which enrolled 19 patients with sHVOD. Give defibrotide 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 defibrotide relatively well. In addition, many trials have confirmed that defibrotide (sodium defibrotide) is safe and effective in the treatment of hepatic veno-occlusive disease (HVOD).

Defibrotide has also been granted "orphan drug" status by the FDA, which can be used not only by adults but also by children. In order to exert the effect of defibrotide in the treatment of hepatic venule occlusion, patients need to take the medication correctly. The recommended dose of defibrotide (defibrotide 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 (sodium defibrotide) is 21 days. If signs and symptoms of HVOD have not resolved after 21 days, continue defibrotide until resolution of HVOD or up to a maximum of 60 days. Use is contraindicated if the patient has active bleeding; monitor the patient for signs of bleeding during treatment. Do not use if patient is allergic to defibrotide or its components.