去纤苷适用于肝小静脉闭塞病的治疗

Suitable for the treatment of hepatic veno-occlusive disease

Hepatic veno-occlusive disease (HVOD) is one of the serious complications after hematopoietic stem cell transplantation (HSCT), and the mortality rate in patients with severe HVOD can be as high as 100%. Defibrotide is a mixture of single-stranded oligonucleotides with antithrombotic and fibrinolytic effects.

In recent years, multiple clinical research results have shown that defibrotide is a safe and effective drug for preventing and treating HVOD after HSCT. Defibrotide was first developed by Gentium Pharmaceuticals in Italy and was approved for marketing in the EU in October 2013. After Gentium Pharmaceuticals was acquired by American Jazz Pharmaceuticals, defibrotide was approved by the U.S. Food and Drug Administration (FDA) for marketing in the United States on March 30, 2016.

Defibrotide is currently recognized as the most promising new drug for the treatment of hepatic veno-occlusive disease (HOVD) after hematopoietic stem cell transplantation (HSCT). Based on its anticoagulant, fibrinolysis-promoting, and anti-inflammatory effects, its application in the treatment of HVOD after HSCT was explored. This study included 19 sHOVD patients. 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 defibrotide well. Since then, multiple clinical trials have confirmed the effectiveness of defibrotide in the treatment of HVOD.

In addition, the most common side effects (incidence ≥10% and independent causal relationship) with defibrotide treatment are hypotension, diarrhea, vomiting, nausea, and epistaxis.

The incidence rate of related grade 3-4 reactions was 7%, mainly hypotension, pulmonary and gastrointestinal bleeding, and abdominal cramps. Only 4% of patients stopped treatment due to side effects. In other words, the side effects of defibrotide are usually mild, and patients do not need to worry too much.

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