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(Alias: defibrotide) is a mixture of single-stranded oligonucleotides broken down from genomic DNA from calf lung or pig intestinal mucosa. The oligonucleotide chain length of defibrinated sodium varies, the relative molecular mass is between 15×10³-30×10³, and its purine to pyrimidine ratio is greater than 0.85. The mechanism of action of defibrinated sodium is relatively complex and is not yet fully understood. Preclinical tests have confirmed that defibrinated sodium has anti-thrombotic, fibrinolytic, anti-ischemic, and anti-infective effects in vivo and in vitro.

Defibrotide sodium (defibrinoside) 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 Jazz Pharmaceuticals of the United States, defibrotide was approved by the U.S. Food and Drug Administration (FDA) for marketing in the United States on March 30, 2016, under the trade name Defitelio.

The effect of defibrinated sodium in the treatment of patients with hepatic veno-occlusive disease:

In a phase III multi-center randomized clinical trial completed in Europe, a total of 356 stem cell transplant patients were recruited. The incidence of hepatic vein occlusion 30 days after transplantation was compared. It was found that 2 of 180 patients in the defibrinated sodium group developed hepatic vein occlusion, accounting for 12%; 35 of 176 patients in the control group developed hepatic vein occlusion, accounting for 20%.

The effectiveness of defibrinated sodium was evaluated through three research trials on 528 patients. The subjects participating in the three trials were all patients diagnosed with VOD symptoms after HSCT and accompanied by abnormal liver or renal function. The overall survival rate 100 days after HSCT was used as the evaluation index.

The results showed that the survival rate after 100 days for patients treated with defibrotide was 38% to 45%; while data analysis found that the survival rate after 100 days for HSCT patients who were expected to receive only supportive care or other drug intervention was only 21% to 31%.

In summary, the treatment of hepatic veno-occlusive disease is very effective and provides a new treatment option for such patients.