去纤苷治疗肝小静脉闭塞病患者效果好吗？

Defibrotide, also called defibrotide sodium, was first developed by Gentium Pharmaceuticals in Italy and was later acquired by Jazz Pharmaceuticals in the United States. Defibrotide was approved for marketing in the European Union in October 2013, and was approved for marketing in the United States by the U.S. Food and Drug Administration (FDA) on March 30, 2016.

Defibrotide has not yet been marketed in my country, and many patients are not familiar with this drug. Today we will mainly look at the effect of defibrotide in the treatment of patients with hepatic veno-occlusive disease.

As the first FDA-approved drug to treat severe hepatic veno-occlusive disease, defibrotide can not only be used for adult patients with hepatic vein occlusion (VOD) who have undergone blood or bone marrow hematopoietic stem cell transplantation (HSCT) and are accompanied by renal or pulmonary function abnormalities, it is also effective in treating pediatric patients with this symptom. The drug has also obtained "Orphan Drug" designation from the FDA.

The effect of defibrotide (sodium defibrotide) in the treatment of hepatic veno-occlusive disease was verified in a large series of prospective clinical studies. The trial included 305 patients with VOD after HSCT, including 220 patients with sHVOD. Give defibrinoside 25 mg/kg per day by intravenous drip. The median age of patients was 16 years old (0.1-70 years old).

Preliminary results show that the CR (complete response) rate is 30%, and the OS (overall survival rate) 100 days after transplantation is 50%; the CR rate of sHVOD patients is 26%, and the OS rate 100 days after transplantation is 45%; the CR rate of non-sHVOD patients is 39%, and the OS rate 100 days after transplantation is 65%. The CR rate of pediatric patients was 33%, and the overall survival rate 100 days after transplantation was 56%; the CR rate of adult patients was 26%, and the overall survival rate 100 days after transplantation was 44%.