去纤维钠的治疗效果怎么样？

(Defitelio) is a polydisperse mixture of single-stranded oligonucleotides that can prevent endothelial cell damage without increasing the risk of systemic bleeding, and protect liver sinusoidal endothelial cells without weakening the anti-tumor effect of cytotoxic drugs. The drug is not only used to prevent hepatic veno-occlusive disease (VOD), but can also be used to treat hepatic veno-occlusive disease (HVOD) and has good efficacy, and side effects such as nausea, hypotension, fever and severe bleeding are rare.

A multicenter phase II randomized study divided 150 HVOD patients into 2 groups, who were treated with defibrinated sodium 25 mg/kg (group A) and 40 mg/kg (group B) every day respectively. The median treatment time was 19 (1-82) days. Results: 141 patients were evaluable for efficacy: 65 patients achieved complete remission (46%), and 62 patients were still alive at 100 days (41%). Although grade 3/4 events in group B patients included increased bleeding and hypotension, the difference was not statistically significant compared with the patients in group A, indicating that high-dose defibrinated sodium had smaller side effects and defibrinated sodium was safe and effective in the treatment of HVOD.

There is no significant difference in efficacy between 25 mg/kg and 40 mg/kg doses of defibrinated sodium per day. Increasing the dose has a tendency to increase toxicity. Therefore, the best way to treat HVOD with defibrinated sodium may be a low dose of 25 mg/kg per day.

The survival status of 80 patients who underwent HSCT was retrospectively analyzed. As a result, 14 patients developed HVOD (4 cases were mild HVOD, 4 cases were moderate HVOD, and 6 cases were severe HVOD). All of them were treated with defibrotide sodium, with a median time of 21.5 ( After 4 to 39) days, 3 patients with severe and all mild and moderate HVOD achieved complete remission (CR), and 14 patients survived 100 days after HSCT. The overall effective rate was 78.56%, while the CR rate among patients with severe HVOD reached 50%. In addition, (defibrinoside) has also achieved promising results in the treatment of severe HVOD in children.

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