去纤苷对肝静脉闭塞病的效果

Effect on hepatic veno-occlusive disease:

In a major study involving 102 patients with severe VOD after blood stem cell transplantation, defibrotide was compared with a historical control group of patients who received standard supportive care. Defibrotide was shown to reduce mortality to 100% in 62% of patients a few days after transplantation, and 24% of patients had complete resolution of symptoms of severe VOD after 100 days.

The benefits of defibrotide also emerged in U.S. patient registry data, in which patients with severe VOD undergoing blood stem cell transplantation who received defibrotide plus standard care had better outcomes than those who received standard care alone, including higher 100-day survival rates (39% vs. 31%) and a higher proportion of patients with resolution of VOD (51% vs. 29%).

The U.S. FDA's approval of defibrotide was based on the results of three clinical studies (Phase 2, Phase 3, and Expanded Access Study) in patients with hepatic VOD and renal or pulmonary insufficiency. Subjects were treated with 6.25 mg/kg of defibrotide every six hours, administered as a two-hour intravenous infusion.

The phase 2 study included 75 adult and pediatric patients with multiple organ dysfunction of the liver (kidney or lung or both) who received defibrinoside (defibrinosodium) for 14 days. The observed survival rate in the trial over 100 days after HSCT was 44%.

The phase 3 study enrolled 102 patients with liver VOD. The treatment group was given the drug for a minimum of 21 days, and patients in this group were not allowed to receive any concomitant drugs, such as heparin, warfarin or alteplase to avoid the risk of bleeding. 

The observed survival rate at +100 days after HSCT was 38%. In the expanded access study, which enrolled 351 patients with liver VOD with hepatorenal disease or pulmonary insufficiency, the survival rate at +100 days after HSCT was 45%. The safety and effectiveness were evaluated in a study involving 176 patients in a clinical development program for the treatment of VOD.

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