去纤苷对肝小静脉闭塞病的效果

(Alias: defibrinated sodium) Therapeutic effect on hepatic veno-occlusive disease:

Defitelio 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.

Defibrotide is the first drug approved by the U.S. FDA to treat severe hepatic veno-occlusive disease. It is used for adult or pediatric patients with hepatic venous obstruction (VOD) who have undergone blood or bone marrow hematopoietic stem cell transplantation (HSCT) and are accompanied by renal or lung function abnormalities. The drug has also received "orphan drug" designation.

Defibrotide (alias: defibrotide sodium) 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 the inflammatory damage of endothelial cells.

In clinical trials, heparin was used to prevent diseases such as hepatic veno-occlusion while greatly increasing the risk of bleeding, while defibrinoside alone or in combination with heparin had a very low incidence of side effects and achieved good results in preventing HVOD.

In a phase III multi-center randomized clinical trial completed in Europe, a total of 356 stem cell transplant patients were recruited. The incidence of hepatic vein occlusion 30 days after transplantation was compared. It was found that 2 of 180 patients in the defibrinated sodium group developed hepatic vein occlusion, accounting for 12%; 35 of 176 patients in the control group developed hepatic vein occlusion, accounting for 20%.

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