去纤苷能有效治病吗？

Introduction: Defibrotide, a mixture of single-stranded oligonucleotides purified from porcine intestinal mucosa, is used to treat hepatic veno-occlusive disease in humans undergoing bone marrow transplantation, with different restrictions in the United States and the European Union. Defibrotide works by protecting the cells lining blood vessels in the liver and preventing blood clotting, in a manner that is not yet understood.

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

Clinical trial data show that it is significantly effective in preventing and treating hepatic veno-occlusive disease. However, there is currently no generic version of defibrotide on the market, and its therapeutic effect cannot be known. Typically, generic drugs need to undergo strict quality control and bioequivalence studies to ensure their consistency with the original drugs. The efficacy and safety of generic drugs that have not gone through these procedures cannot be guaranteed.

The original drug of defibrotide has proven its effectiveness through clinical trials and has received official approval. Whether the generic version is effective requires specific clinical data and official certification to support it. Without relevant data information, the effectiveness of counterfeit versions cannot be determined. Patients and medical professionals are advised to use officially approved medications to ensure the safety and effectiveness of treatment.

Efficacy of defibrotide

528 subjects were investigated in three studies, two prospective clinical trials and one expanded access study. Subjects in all three studies were diagnosed with hepatic VOD after hematopoietic stem cell transplantation (HSCT) and had associated hepatic or renal abnormalities. These studies measured the percentage of subjects alive 100 days after HSCT (overall survival rate).

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

Side effects of defibrotide

Defibrotide has a higher risk of bleeding, and some people have an allergic reaction to defibrinogen. Common side effects include impaired coagulation, vomiting, low blood pressure, bleeding in the brain, eyes, lungs, stomach or intestines, bleeding in the urine, and bleeding at the catheterization site. Other side effects include diarrhea, nosebleeds, sepsis, graft-versus-host disease, and pneumonia.

Pregnant women should not use defibrotide, nor should women become pregnant while using this drug. The drug has not been tested in pregnant women, but at normal doses, the drug can cause hemolytic abortion in rats.