肝小静脉闭塞病患者治疗新药-去纤苷

For some patients with leukemia and lymphoma, hematopoietic stem cell transplantation (HSCT) is the only hope to cure the disease and regain health. Hepatic veno-occlusive disease (HVOD) is one of the serious complications after hematopoietic stem cell transplantation (HSCT), and the mortality rate in patients with severe HVOD can be as high as 100%. Defibrotide (defiteli) is a new drug for the treatment of hepatic veno-occlusive disease. Its launch brings a safe and effective solution to the treatment of HVOD patients.

Defibrotide (defiteli) 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.

Based on the anticoagulant, fibrinolysis-promoting, and anti-inflammatory effects of defibrotide (defiteli), Richardson et al. first explored the application of defibrotide in the treatment of HVOD after HSCT. This study included 19 patients with sHVOD. The dosage of defibrotide is 5~60mg/kg per day, intravenous infusion, and the median usage time is 15d (2-61d).

The results showed that 42% of patients achieved complete remission (CR), and the overall survival (os) rate 100 days after transplantation reached 32%, and patients could tolerate defibrotide well. Since then, many clinical trials have confirmed the effectiveness of defibrotide (defiteli) in the treatment of HVOD.

It was approved for marketing in the United States by the U.S. Food and Drug Administration (FDA) on March 30, 2016. Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the Center for Drug Evaluation and Research of the U.S. FDA, said: "The approval of defibrotide fills a huge need. Patients will have rare but often fatal complications after receiving chemotherapy and HSCT, and defibrotide is targeted for this indication."