去纤维钠治疗肝小静脉闭塞病患者的效果

Defibrotide is an adenosine receptor agonist with multiple effects. The adenosine A1/A2 receptors of endothelial cells are involved in the regulation of endothelial cells and the response of endothelial cells to injury. Defibrotide can act on these receptors to produce a variety of downstream effects. Defibrotide can also reduce the expression of cell adhesion molecules on the surface of endothelial cells, thereby reducing the adhesion of leukocytes to vascular endothelial cells and reducing inflammatory damage to endothelial cells. Defibrotide is a polydisperse mixture of single-stranded oligonucleotides that can prevent endothelial cell damage without increasing the risk of systemic bleeding, and protect liver sinusoidal endothelial cells without weakening the anti-tumor effect of cytotoxic drugs. Defibrotide is not only used to prevent hepatic veno-occlusive disease (VOD), but can also be used to treat hepatic veno-occlusive disease (HVOD) with good efficacy, and side effects such as nausea, hypotension, fever and severe bleeding are rare.

The effect of defibrotide in the treatment of patients with hepatic veno-occlusive disease: In one study, 19 patients with sHVOD were given Defibrotide 5~60 mg/kg every day as intravenous infusion treatment, 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 rate reached 32% 100 days after transplantation. Not only that, Defibrotide 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 Defibrotide be used to treat adults, children can also benefit from it.

A multicenter phase II randomized study divided 150 HVOD patients into 2 groups, who were treated with Defibrotide 25 mg/kg (group A) and 40 mg/kg (group B) every day respectively. The median treatment time was 19 (1-82) days. Results: 141 patients were evaluable for efficacy: 65 patients achieved complete remission, and 62 patients were still alive at 100 days. Although grade 3/4 events in patients in group B included increased bleeding and hypotension, the difference was not statistically significant compared with patients in group A, indicating that the side effects of high-dose Defibrotide were smaller and Defibrotide was safe and effective in the treatment of HVOD.

The above is the introduction about Defibrotide, I hope it can help everyone.

Note: The above information comes from the Internet and is compiled and edited by Medical Companion Travel (please correct me if there are any errors or omissions). It is only to provide information on the latest drugs on the market in the world and help Chinese patients understand the latest international new drug trends. It is only for internal discussion among medical staff and does not serve as any basis for medication. For specific medication guidelines, please consult the attending physician. 

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