去纤苷目前已知获批适应症有哪些呢？

Defiteli is approved for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), and renal or pulmonary dysfunction after hematopoietic stem cell transplantation (HSCT).

The recommended dose of defibrotide 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.

Give defibrotide (defiteli) for a minimum of 21 days. If signs and symptoms of hepatic VOD have not resolved after 21 days, continue defibrotide until resolution of VOD or up to a maximum of 60 days. Defibrotide must be diluted prior to infusion.

Defibrotide (defiteli) has the effects of anticoagulation, promoting fibrinolysis, and also promotes vascularization. Basic fibroblast growth factor (bFGF) has the effect of stimulating vascularization. Some studies have found that defibrotide can not only combine with basic fibroblast growth factor (bFGF) to mobilize bFGF from the extracellular matrix, but can also protect bFGF from protease degradation, thereby stimulating angiogenesis.

In a phase 2 trial of 75 patients, a phase 3 trial of 102 patients, and an expansion trial of an additional 351 patients, all patients in the trial were diagnosed with hepatic VOD with renal or pulmonary dysfunction after HSCT. 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 hepatic VOD who were not treated with defiteli was 21%-31%.

The above is an introduction to the indications of defibrotide, I hope it will be helpful to you.