去纤维钠的安全性及疗效

Defibrotide is a mixture of sodium salts of single-stranded deoxyoligonucleotides produced from porcine intestinal mucosal genomic DNA through controlled depolymerization. On March 30, 2016, Defibrotide was approved by the US FDA for the treatment of severe hepatic veno-occlusive disease in adults and children after hematopoietic stem cell transplantation (usually complicated by renal and pulmonary function abnormalities).

Safety and efficacy of Defibrotide: In the pilot study, 528 patients diagnosed with VOD symptoms after HSCT were included in 3 studies to evaluate the effectiveness of Defibrotide from Sir Irish Pharmaceuticals. These patients were also accompanied by liver or kidney function abnormalities. The evaluation index is the overall survival rate 100 days after HSCT.

The results showed that the survival rate of patients treated with Defibrotide after 100 days was 38% to 45%; while data analysis found that the survival rate of HSCT patients who were expected to receive only supportive care or other drug intervention after 100 days was only 21% to 31%. The use of Defibrotide can effectively improve the survival rate of patients. In clinical trials, heparin is usually used to prevent diseases such as hepatic vein occlusion, which also greatly increases the patient's risk of bleeding. Defibrotide alone or in combination with heparin has a low incidence of side effects, good safety, and has achieved good VOD prevention effects. Defibrotide is also currently recognized as a relatively effective drug in the treatment of hepatic veno-occlusive disease (VOD).

Treatment with Defibrotide will also produce certain side effects or adverse reactions. The most common side effects of this drug include: hypotension/diarrhea/vomiting/nausea/nose bleeding, etc. The incidence rate of grade 3-4 reactions related to defibrotide is 7%, mainly hypotension/pulmonary and gastrointestinal bleeding/abdominal cramps, etc. The side effects of Defibrotide are usually mild, and patients do not need to worry too much. Concomitant use of Defibrotide and a systemic anticoagulant or fibrinolytic therapy may increase the risk of bleeding. Discontinue anticoagulants and fibrinolytics before defibrotide treatment, and consider delaying initiation of defibrotide administration until the effects of the anticoagulant have lessened.

The above is the introduction about Defibrotide. If patients have other questions about Defibrotide (such as drug price, purchase, etc.), they can consult the medical companion travel service.

Note: The above information comes from the Internet and is compiled and edited by Medical Companion Travel (please correct me if there are any errors or omissions). It is only to provide information on the latest drugs on the market in the world and help Chinese patients understand the latest international new drug trends. It is only for internal discussion among medical staff and does not serve as any basis for medication. For specific medication guidelines, please consult the attending physician. 

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