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去纤苷治疗效果怎么样?

Author: Medicalhalo
Release time: 2025-10-19 11:44:20

Therapeutic effects of treating hepatic veno-occlusive disease:

Richardson et al first explored its application in the treatment of H hepatic veno-occlusive disease after HSCT. This study included 19 patients with hepatic veno-occlusive disease. The dosage of defibrotide is 5~60mg/kg per day, intravenous infusion, and the median usage time is 15d (2-61d). The results showed that 42% of patients achieved complete remission (CR), and the overall survival (os) rate 100 days after transplantation reached 32%, and patients could tolerate defibrination well. Since then, multiple clinical trials have confirmed the effectiveness of defibrotide in the treatment of hepatic veno-occlusive disease.

Additionally, the FDA's approval of defibrotide underscores the importance of defibrotide in children and adults as the first and only proven treatment for this rare and often fatal complication of stem cell transplantation.

Defibrotide is a clinically significant therapeutic advance because it represents a potentially curative intervention for patients with hepatic veno-occlusive disease, which may save lives over a course of treatment.

The FDA approval of defibrotide is supported by efficacy data from three clinical studies of defibrotide 6.25 mg/kg every 6 hours in patients with hepatic veno-occlusive disease and renal or pulmonary dysfunction after liver transplantation.

The safety profile supporting the approval of defibrotide was based on data from a clinical development program in 176 patients who underwent defibrotide-treated HSCT for the treatment of hepatic veno-occlusive disease with renal and/or pulmonary dysfunction.

The most common adverse reactions (occurring in 10% and not related to cause and effect) with defibrotide treatment are hypotension (low blood pressure), diarrhea, vomiting, nausea, and epistaxis (nose bleeding).

The most common serious adverse reactions (incidence 5%, independent of causality) were hypotension (11%) and alveolar hemorrhage (7%).

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