去纤维钠对肝小静脉闭塞病有效果吗？

(Defibrinoside) is the first drug approved by the U.S. FDA to treat severe hepatic veno-occlusive disease. It is used for adult or pediatric patients with hepatic venous obstruction (VOD) who have undergone blood or bone marrow hematopoietic stem cell transplantation (HSCT) and are accompanied by renal or pulmonary function abnormalities. The drug has also received "orphan drug" designation.

Hepatic veno-occlusive disease (VOD) is one of the serious complications after hematopoietic stem cell transplantation (HSCT), and the mortality rate in patients with severe hepatic veno-occlusive disease (VOD) can be as high as 100%. Defibrotide sodium 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 sodium is a safe and effective drug for preventing and treating VOD after HSCT.

Defibrotide sodium (defibrotide) is not currently on the market in my country. For domestic patients, this drug is relatively unfamiliar, and what everyone wants to know most is its therapeutic effect.

A Phase 2 trial with 75 patients, a Phase 3 trial with 102 patients, and an expansion trial with 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%.

(Defibrinoside) is an adenosine receptor agonist with multiple effects. Correct use can ensure the effectiveness. It needs to be carried out under the guidance of a professional doctor during use. The recommended dose for adult and pediatric patients is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. The dose should be based on the patient's baseline weight, which is defined as the patient's weight before the HSCT preparation protocol.