肝小静脉闭塞病治疗新药去纤苷治疗效果怎么样？

It is a mixture of single-stranded oligonucleotides with anti-thrombotic and fibrinolysis-promoting effects. In recent years, multiple clinical research results have shown that defibrotide (defibrotide sodium) is a safe and effective drug for preventing and treating HVOD after HSCT. Today we will learn in detail about the therapeutic effect of defibrotide, a new drug for the treatment of hepatic veno-occlusive disease.

Defibrotide (Defibrotide Sodium) is the first drug approved by the FDA for the treatment of severe hepatic venular occlusion, a rare and fatal liver disease. The approval was based on a Phase 2 trial of 75 patients, a Phase 3 trial of 102 patients, and an expansion trial of an additional 351 patients.

All patients in the trial were diagnosed with hepatic veno-occlusive disease and associated renal or pulmonary dysfunction after HSCT (hematopoietic stem cell transplantation). In the Phase 2 trial, 44% of patients survived 100 days after transplantation, compared with 38% in the Phase 3 trial, and 45% in the expanded trial, and the FDA noted in a report that the expected 100-day survival rate for patients with severe veno-occlusive disease who were not treated with defibrotide was 21%-31%.

In a pilot study, 528 patients diagnosed with VOD symptoms after HSCT were included in 3 studies to evaluate the effectiveness of defibrotide (defibrotide sodium). These patients also had abnormal liver or renal function. The evaluation index is the overall survival rate 100 days after HSCT.

The results showed that the survival rate of patients who received defibrotide treatment after 100 days was 38% to 45%; while data analysis found that the survival rate of HSCT patients who were expected to receive only supportive care or other drug intervention after 100 days was only 21% to 31%. The use of defibrotide can effectively improve the survival rate of patients.

In clinical trials, heparin is usually used to prevent diseases such as hepatic veno-occlusion, which also greatly increases the patient's risk of bleeding. However, defibrotide alone or in combination with heparin has a low incidence of side effects, good safety, and has achieved good VOD prevention effects. Defibrotide (defibrotide sodium) is also currently recognized as the most promising drug for the treatment of hepatic veno-occlusive disease (VOD).

In 2002, a multi-center collaborative group in the United States reported the results of using defibrotide in 87 patients with severe HVOD and multiple organ failure after hematopoietic stem cell transplantation. The dosage was 5-60 mg/kg/d, and the median duration of medication was 15 days. As a result, 36% of the patients achieved complete remission, and 35% of the patients survived 100 days after transplantation. Both studies showed that defibrotide has no obvious toxic side effects and is a safe and effective drug for the treatment of HVOD.

The above is the content of the therapeutic effect of (Defibrinated Sodium), I hope it can help you!

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