去纤维钠治疗肝小静脉闭塞病疗效如何？

(Alias: Defibrotide) is indicated for adults and children with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), and renal or pulmonary dysfunction after hematopoietic stem cell transplantation (HSCT). Defibrotide sodium was first developed by Gentium Pharmaceuticals in Italy and was approved for marketing in the EU in October 2013.

The recommended dose of defibrotide sodium is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. Treatment lasts for a minimum of 21 days. If signs and symptoms of VOD do not resolve after 21 days, continue treatment until resolution. Defibrinated sodium must be diluted before infusion. Prior to administration of defibrinated sodium, confirm that the patient has not experienced clinically significant bleeding and is hemodynamically stable on no more than one vasopressor.

How effective is defibrinated sodium in the treatment of hepatic veno-occlusive disease?

Studies have shown that defibrotide sodium (defibrinoside) mainly exerts various biological functions by affecting vascular endothelial cell response and platelet activity. Defibrinated sodium contains nucleic acid aptamers that interact with adenosine A1 and A2 receptors, thereby reducing the response of endothelial cells to injury and protecting vascular endothelium. In vitro experiments and in vivo animal experiments have confirmed that defibrinated sodium can stimulate the release of prostacyclin PGI2. PGI2 is mainly synthesized and secreted by vascular endothelium and platelets. It dilates blood vessels and inhibits platelet aggregation by increasing the level of cAMP. Elevated PGI2 can also inhibit the lipoxygenase pathway to reduce the production of leukotriene LTB4 and reduce ischemia-induced tissue damage.

In one study, 19 patients with sHOVD were included. (Defibrinoside) dosage is 5~60 mg/kg per day, intravenous infusion, and 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 (os) rate 100 days after transplantation reached 32%, and the patients could tolerate defibrinated sodium well. Since then, multiple clinical trials have confirmed the effectiveness of defibrotide in the treatment of hepatic veno-occlusive disease.

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