去纤苷在中国怎么买？

On March 30, 2017, it was granted priority review and orphan drug status by the US FDA. The approval was based on 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 and associated 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 in patients with severe hepatic VOD who were not treated with defibrotide was 21%-31%. Data on the drug's safety are based on 176 patients. The most common adverse reactions include hypotension, diarrhea, vomiting, nausea, and epistaxis. The most common serious adverse reactions included hypotension (11%) and alveolar hemorrhage (7%). The approval of defibrotide greatly meets the treatment needs of transplant patients suffering from such rare and fatal complications.

The researchers recruited a total of 356 subjects during three years (2006.1.25-2009.1.29). There are 28 European university hospitals and medical schools participating. Participants were under 18 years old and signed informed consent forms. Subjects who have received myeloablative conditioning before allogeneic or allogeneic hematopoietic stem cell transplantation, and at the same time, according to the "Seattle Criteria Revised", the subject has single or multiple veno-occlusive disease susceptibility factors. The subjects were randomly assigned by computer into two groups: prevention group (intravenous administration) and control group (placebo). The primary endpoint was the incidence of veno-occlusive disease in subjects within 30 days after hematopoietic stem cell transplantation, and side effects that occurred within 180 days after transplantation in the prevention group were observed. If the subject develops advanced veno-occlusive disease, the experiment will be discontinued and treatment will be provided. The results showed that within 30 days after hematopoietic stem cell transplantation, the incidence of veno-occlusive disease in the prevention group was 12% (22/180), and that in the control group was 20% (35/176); the risk difference between the two groups was 7.7%, Z test p=0.0488, the difference was statistically significant At the same time, 87% (154/177) of the prevention group and 88% (155/176) of the control group had side effects within 180 days after transplantation. There was no difference, indicating that the side effects were caused by blood stem cell transplantation or veno-occlusive disease, not fibrinide.

The only fly in the ointment is that defibrotide has not been approved for marketing in China. If domestic patients want to obtain defibrotide, they can purchase it in person in countries that have approved it for sale, or they can obtain it through domestic professional overseas medical service institutions.