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肝小静脉闭塞病新药--去纤苷

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

The latest new drug produced: What about the new drug for hepatic veno-occlusive disease - defibrotide? Is it effective? Let me introduce it to you below. Defibrotide (defiteli) is a mixture of single-stranded oligonucleotides broken down from genomic DNA from calf lung or porcine intestinal mucosa. The oligonucleotide chain lengths of defiteli (sodium defiteli) vary, the relative molecular mass ranges from 15×10³-30×10³, and its purine to pyrimidine ratio is greater than 0.85. The mechanism of action of defibrotide (defiteli, defiteli) is relatively complex and is not yet fully understood. Preclinical trials have confirmed that defibrinoside (defiteli, defiteli) has anti-thrombotic, fibrinolytic, anti-ischemic, and anti-infective effects in vivo and in vitro.

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 (defiteli) is a single-stranded oligonucleotide mixture that has anti-thrombotic and fibrinolytic effects. In recent years, multiple clinical research results have shown that defibrotide (defiteli) is a safe and effective drug for preventing and treating HVOD after HSCT.

A large series of prospective clinical studies included 305 patients with HVOD after HSCT, with a median age of 16 years (0.1-70 years), including 220 patients with sHVOD. The therapeutic dose of defibrotide (defiteli, defiteli) is 25 mg/kg per day, intravenous infusion.

Preliminary results show that: the complete response (CR) rate is 30%, and the overall survival (OS) rate 100 days after transplantation is 50%; the CR rate of sHVOD patients is 26%, and the OS rate 100 days after transplantation is 45%; the CR rate of non-sHVOD patients is 39%, and the OS rate 100 days after transplantation is 65%. The CR rates of pediatric and adult patients were 33% and 26%, respectively (P=0.187), and the OS rates 100 days after transplantation were 56% and 44%, respectively (P=0.0277).

Defibrotide (defiteli) is a very effective drug for the treatment of hepatic veno-occlusive disease. Patients can buy it with confidence. For specific questions, please consult your medical companion.

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