去纤苷治疗效果怎样？

Defitelio (sodium defibrate) was first developed by Gentium Pharmaceuticals in Italy 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 indicated for the treatment of 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 is a relatively unfamiliar drug to domestic patients, so what is its therapeutic effect?

Defibrotide (sodium 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 the inflammatory damage of endothelial cells.

In a phase 2 trial of 75 patients, a phase 3 trial of 102 patients, and an expansion trial of an additional 351 patients, all patients in the trial were diagnosed with hepatic VOD with renal or pulmonary dysfunction after HSCT. In the phase 2 trial, 44% of patients survived 100 days after transplantation, compared with 38% in the phase 3 trial and 45% in the expanded trial, and the FDA noted in a report that the expected 100-day survival rate for patients with severe hepatic VOD who were not treated with defibrotide (desoside) was 21%-31%.

Hypersensitivity reactions have occurred in less than 2% of patients treated with defibrotide. These reactions include rash, urticaria, and angioedema. Especially if there is a history of previous exposure. If a severe hypersensitivity reaction occurs, discontinue defibrination, treat with standard medical care, and monitor until symptoms resolve.