去纤苷治疗肝小静脉闭塞病疗效如何？

How effective is defibrinoside (defibrotide sodium) in the treatment of hepatic veno-occlusive disease? Hepatic veno-occlusive disease (HVOD), also known as sinusoidal obstruction syndrome (SOS), is one of the serious complications after hematopoietic stem cell transplantation (HSCT). It mostly occurs within 30 days after HSCT. Its typical clinical manifestations are hepatomegaly, jaundice, weight gain, and abdominal effusion. The incidence of HVOD after HSCT is approximately 8%-14%, and can reach 60% in high-risk patients. The prognosis of patients with severe HVOD (sHVOD) is extremely poor, often complicated by multi-organ failure (MOF), and the mortality rate can be as high as 100%. The treatment of HVOD is still mainly supportive treatment, and there is a lack of effective treatment methods. In recent years, a large number of clinical studies have confirmed the effectiveness of defibrotide (defibrotide sodium) in preventing and treating HVOD.

In clinical trials, while heparin was used to prevent diseases such as hepatic veno-occlusion, it greatly increased the risk of bleeding. However, defibrotide (defibrotide sodium) alone or in combination with heparin had a very low incidence of side effects and achieved good results in preventing HVOD. 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 defibrotide group developed hepatic vein occlusion, accounting for 12%; 35 of 176 patients in the control group developed hepatic vein occlusion, accounting for 20%.

Corbacioglu et al. analyzed the value of defibrotide in the treatment of HVOD in children in a retrospective clinical study, which included 45 patients with HVOD after HSCT who were <20 years old, with a median age of 8.2 years (0.2-20 years). The dose of defibrotide is 10~110 mg/kg per day, intravenous infusion, and the median use time is 17 days (1-83 days). The results showed that the complete response (CR) rate was 76%, and the overall survival (OS) rate 1,00 days after transplantation was 64%. Among them, the CR rate of 22 SHVOD patients was 50%, and the OS rate 100 days after transplantation was 36%. This study not only confirmed the effectiveness of defibrotide (defibrotide sodium) in the treatment of HVOD in children, but also showed that the addition of other drugs for the treatment of HVOD does not affect the efficacy of defibrotide in the treatment of HVOD.