去纤苷治疗肝小静脉闭塞病的疗效怎么样？

It is suitable 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). So what is the efficacy of defibrotide in treating hepatic veno-occlusive disease?

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

Hepatic venule occlusion causes edema and reduced blood flow in the liver due to blockage of some blood vessels in the liver. This condition can lead to the most severe liver damage. The drug is the first drug approved by the FDA for the treatment of severe hepatic venous 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).

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