去纤苷针对肝窦阻塞综合征患者的疗效和安全性

Sinusoidal obstruction syndrome (SOS), formerly known as hepatic veno-occlusive disease (VOD), is a complication after hematopoietic stem cell transplantation (HSCT). Its main clinical manifestations include jaundice, hepatomegaly, ascites, etc. In severe cases, multiple organ failure and even death may occur. The clinical manifestations of SOS/VOD vary in severity. Mild cases can recover within a few weeks, while severe cases can cause multiple organ failure and a mortality rate of over 80%. (defibrotide) is currently the only drug that has been proven to have clear efficacy in the treatment and prevention of SOS/VOD in multiple clinical trials.

In multiple phase I/II clinical trials of defibrotide in SOS/VOD patients (including many patients with multiple organ failure), the complete response rate was 36% to 55%, and the survival rate on the 100th day after transplantation was approximately 32% to 44%. In a historically controlled international multi-center phase III clinical trial, researchers observed defibrotide treatment for VOD/SOS in 102 adults and children (25 mg/kg/day, administered in 4 divided doses). The survival rate of the DF group was 38.2%, while that of the control group was 25% (P=0.0109). At the same time, many other multi-center clinical trials have confirmed the effect of defibrotide in the prevention of SOS/VOD.

There are no unified guidelines for the dosage and duration of treatment of defibrotide. According to foreign experience, intravenous administration should be administered approximately every 6 hours. The initial dose is 6.25 mg/kg. Subsequent doses are increased to 7.5 mg/kg (second dose), 10 mg/kg (third dose), 12.5 mg/kg (fourth dose), and then maintained at 15 mg/kg/time (60 mg/kg/d), and used every 6 hours. Once any significant and reversible reaction occurs, adjust the dose immediately (stop increasing the dose or reduce the dose). In terms of usage time, the planned treatment time is 14 days, but the usage time can be extended or shortened based on clinical needs.

Note: The above information comes from the Internet and is compiled and edited by Medical Companion Travel (please correct me if there are any errors or omissions). It is only to provide information on the latest drugs on the market in the world and help Chinese patients understand the latest international new drug trends. It is only for internal discussion among medical staff and does not serve as any basis for medication. For specific medication guidelines, please consult the attending physician.