去纤苷在治疗肝小静脉闭塞病这方面的效果如何呢？

The U.S. Food and Drug Administration (FDA) has approved it for the treatment of hepatic veno-occlusive disease (VOD) secondary to hematopoietic stem cell transplantation (HSCT) in adults and children.

Defibrotide is the first drug approved to treat severe hepatic veno-occlusive disease. So how effective is defibrotide in the treatment of hepatic veno-occlusive disease?

The therapeutic effect of defibrotide in the treatment of hepatic veno-occlusive disease:

Hepatic veno-occlusive disease is a fatal disease that usually occurs without warning after HSCT, progresses rapidly, and can cause severe renal or pulmonary dysfunction and multi-organ failure. Therefore, this lesion can hinder the recovery of transplant patients, and once hepatic veno-occlusive disease or multiple organ failure occurs, the mortality rate is as high as more than 80%.

Defibrotide was granted priority review and orphan drug designation. The approval was based on 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 veno-occlusive disease and associated 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 veno-occlusive disease who were not treated with defibrotide was 21%-31%.

Data on the drug's safety are based on 176 patients. The most common adverse reactions include hypotension, diarrhea, vomiting, nausea, and epistaxis. The most common serious adverse reactions included hypotension (11%) and alveolar hemorrhage (7%). The approval greatly meets the treatment needs of transplant patients suffering from such rare and fatal complications.

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