去纤维钠治疗肝小静脉闭塞病的效果好吗？

(Defibrotide) is currently recognized as the most promising new drug for the treatment of hepatic veno-occlusive disease (HVOD) after hematopoietic stem cell transplantation (HSCT). Defibrinated sodium has anticoagulant and fibrinolysis effects, and also promotes vascularization. Basic fibroblast growth factor (bFGF) has the effect of stimulating vascularization. Some studies have found that defibrinated sodium can not only combine with basic fibroblast growth factor (bFGF) to mobilize bFGF from the extracellular matrix, but can also protect bFGF from protease degradation, thereby stimulating angiogenesis.

Defiberite sodium was first developed by Italy's Gentium Pharmaceuticals and was approved for marketing in the EU in October 2013. It was approved for marketing in the United States by the U.S. Food and Drug Administration (FDA) on March 30, 2016, under the trade name Defitelio.

The effect of defibrinated sodium in the treatment of hepatic veno-occlusive disease:

In one study, 19 patients with sHVOD were given 5 to 60 mg/kg of defibrotide sodium (defibrinoside) as intravenous infusion every day. The median use time was 15 days (2 to 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.

In a clinical trial on the treatment of HVOD in children, the complete response (CR) rate using defibrotide was 76%, and the overall survival (OS) rate 1,00 days after transplantation was 64%. The effect is very significant. Not only can defibrinated sodium be used to treat adults, children can also benefit from it.

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