去纤维钠的适应症有什么？

(Defibrinoside, defiteli) is a mixture of single-stranded oligonucleotides with anti-thrombotic and fibrinolytic effects. It was approved for marketing in the European Union in October 2013 and approved for marketing in the United States by the U.S. Food and Drug Administration (FDA) on March 30, 2016. It is used for adult or pediatric patients with hepatic vein obstruction (HVOD) and renal or lung function abnormalities after receiving blood or bone marrow hematopoietic stem cell transplantation (HSCT).

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. In recent years, a large number of clinical studies have confirmed the effectiveness of defibrinated sodium in preventing and treating HVOD.

The recommended dose of defibrotide sodium (defiteli) for adult and pediatric patients is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. Administer defibrinated sodium for a minimum of 21 days. If signs and symptoms of hepatic VOD have not resolved after 21 days, continue defibrination until resolution of VOD or up to a maximum of 60 days.

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 was compared 30 days after transplantation. It was found that 2 of 180 patients in the defibrotide (defibrotide) group developed hepatic vein occlusion, accounting for 12%; 35 of 176 patients in the control group developed hepatic vein occlusion, accounting for 20%.

(Defibroside, defiteli) has played a positive role in the treatment of hepatic vein obstruction (HVOD), and has also been awarded the title of "Orphan Drug" by the US FDA.