去纤苷治疗肝小静脉闭塞病患者疗效怎么样？

Defibrotide sodium is also called defibrotide, and its English name is defiteli. It was first developed by Gentium Pharmaceuticals in Italy and was launched in the EU in 2013. Defibrotide sodium is a good medicine for the treatment of hepatic veno-occlusive disease (HVOD), also known as sinusoidal obstruction syndrome (SOS). It can be used not only to treat adults, but children can also benefit from it.

Defibrotide (Defibrotide Sodium) is an injectable formulation and is dosed based on the patient's weight. The recommended dose for adult and pediatric patients is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. The minimum administration time is 21 days. If signs and symptoms of hepatic veno-occlusive disease have not resolved after 21 days, continue defibrotide until resolution of hepatic veno-occlusive disease or for a maximum of 60 days.

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

Richardson et al first explored the application of defibrotide in the treatment of HVOD after HSCT. In this trial, a total of 19 patients with sHVOD participated in the treatment. Defibrinoside was administered at 5~60 mg/kg per day, intravenously infused, 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 severe hepatic VOD who are not treated with defibrotide is 21%-31%. Therefore, the use of defibrotide effectively prolongs the survival time of patients.