去纤苷能有效治病吗?

Introduction: Defibrotide is used to treat adults and children with hepatic veno-occlusive disease (VOD; blockage of blood vessels in the liver, also known as sinus obstruction syndrome) who develop kidney or lung problems after receiving a hematopoietic stem cell transplant. Defibrinosides belong to a class of drugs called antithrombotics, which work by protecting endothelial cells and reducing inflammation and thrombosis to improve a patient's condition.

Can the generic version of defibrotide also be effective in treating diseases?

As of now, we are not aware of the existence of a generic version, so we cannot know its specific efficacy. Generally, generic drugs need to undergo rigorous bioequivalence studies to ensure that they are consistent in quality and efficacy with the original drugs. If there is a generic version of defibrotide on the market in the future, it usually means that its effectiveness and safety have been approved by relevant departments for marketing. Its efficacy is basically the same as that of the original drug, and it can also effectively treat diseases. However, whether it is effective still needs to be determined based on the approval of the drug regulatory authorities and the results of clinical trials of the drug. Therefore, before using any medication, especially generic medications, patients should consult a physician or medical professional to ensure safety and effectiveness.

The therapeutic effect of defibrotide

Three studies including a total of 528 subjects receiving defibrotide were evaluated to evaluate its efficacy, two prospective clinical trials and one expanded access study. The subjects in these three studies were diagnosed with hepatic VOD after hematopoietic stem cell transplantation (HSCT) and were accompanied by liver or kidney abnormalities. These studies measured the percentage of subjects alive 100 days after HSCT (overall survival).

Across the three studies, 38%-45% of subjects treated with defibrotide survived 100 days after HSCT. Based on published reports and analysis of subject-level data, the expected survival rate at 100 days after HSCT for subjects with severe hepatic VOD who receive only supportive care or interventions other than fiber removal is 21%-31%.

Side effects of defibrotide

Defibrotide carries a higher risk of bleeding, and some people have allergic reactions to defibrotide. Common side effects of defibrotide include impaired coagulation, vomiting, hypotension, and bleeding in the brain, eyes, lungs, stomach, or intestines, bleeding in the urine, and bleeding at the catheterization site. Also included are diarrhea, nosebleeds, sepsis, graft-versus-host disease, and pneumonia.