How effective is defibrotide in hepatic veno-occlusive disease?

How effective is defibrotide in hepatic veno-occlusive disease? Defibrinoside sodium is a polydeoxyribose ribonate that has certain fibrinolytic and antithrombotic activities. It can promote the production of prostacyclin-like substances and release them from vascular tissues into the circulation. This medicine is indicated for the treatment of adult and pediatric patients with hepatic venous occlusion (VOD) and associated renal or pulmonary abnormalities after blood or bone marrow hematopoietic stem cell transplantation (HSCT).

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 sodium greatly meets the treatment needs of transplant patients suffering from such rare and fatal complications.

Defibrotide sodium is currently the only FDA-approved treatment for adult and pediatric patients who develop hepatic venule occlusion (VOD) and associated 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 sodium significantly improves survival after 100 days. The above is the therapeutic effect of defibrotide, I hope it can help you.