去纤维钠疗效怎么样？

It is an adenosine receptor agonist with multiple effects. It was approved for marketing in the United States in 2016 and is known as an "orphan drug". It is also the first drug approved by the US FDA to treat severe hepatic venous obstruction. It was developed by Gentium Pharmaceuticals (later acquired by Jazz Pharmaceuticals of the United States). It is also known as defiteli.

Because defibrinated sodium has not yet been launched in my country, many patients are unfamiliar with it. But what everyone wants to know most is the efficacy of defibrinated sodium. Let’s take a look today.

In one study, 19 patients with sHVOD were given intravenous infusion of defibrinated sodium 5 to 60 mg/kg daily, with a median use time of 15 days (2-61 days). In this study, a total of 42% of patients achieved complete response (CR), and the overall survival (OS) rate at 100 days after transplantation was 32%. Not only that, defibrinated sodium is also better tolerated by patients. Since then, multiple clinical trials have confirmed the effectiveness of defibrotide in the treatment of hepatic veno-occlusive disease.

Defibrotide sodium not only plays a positive role in the treatment of adults, but also plays a very important role in children.

Effectiveness in pediatric patients comes from an analysis of a retrospective clinical study. The 45 post-HSCT HVOD patients included in the study were all younger than 20 years old, with a median age of 8.2 years (0.2-20 years). Defibrinated sodium was administered daily at 10~110 mg/kg by intravenous infusion, and the median usage time was 17 days (1-83 days). The results showed that the CR rate was 76%, and the OS rate 1,00 days after transplantation was 64%. Among them, the CR rate of 22 SHVOD patients was 50%, and the OS rate 100 days after transplantation was 36%. This study not only confirmed the effectiveness of defibrotide sodium in treating HVOD in children, but also showed that the addition or absence of other drugs for the treatment of HVOD did not affect the efficacy of defibrotide sodium HVOD.