去纤苷治疗肝静脉闭塞病效果怎么样？

It is suitable for the treatment of adults and children with hepatic veno-occlusive disease (VOD), also known as hepatic sinusoidal obstruction syndrome (SOS), and renal or pulmonary dysfunction after hematopoietic stem cell transplantation (HSCT). How effective is defibrotide in treating hepatic veno-occlusive disease?

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

Defibrotide is the first drug approved by the FDA for the treatment of severe hepatic venule occlusion, a rare and fatal liver disease.

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 VOD 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 in patients with severe hepatic VOD 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 include hypotension and alveolar hemorrhage. The approval of defibrotide greatly meets the treatment needs of transplant patients suffering from such rare and fatal complications.

It is currently the only FDA-approved treatment for adult and pediatric patients with hepatic venous occlusion (VOD) and renal or pulmonary abnormalities after blood or bone marrow hematopoietic stem cell transplantation (HSCT). Although safety data from randomized controlled trials are insufficient, available efficacy data confirm that defibrotide significantly improves survival after 100 days.

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