去纤维钠治疗效果

As the first drug approved by the FDA to treat severe hepatic veno-occlusive disease, (Defibrotide) can not only be used for adult patients with hepatic vein occlusion (HVOD) who have undergone blood or bone marrow hematopoietic stem cell transplantation (HSCT) and are accompanied by renal or pulmonary function abnormalities, but also has a therapeutic effect on pediatric patients with this symptom. This drug has also obtained "Orphan Drug" designation from the FDA.

Defibrotide sodium is dosed primarily based on patient weight. The recommended dose for adults and children is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. Patients should start using it after diagnosis by a doctor with treatment experience. Blind use of medication may cause harm to themselves.

The therapeutic effect of defibrinated sodium:

Richardson et al first explored the application of defibrinated sodium (defibrotide) in the treatment of hepatic vein occlusion after hematopoietic stem cell transplantation (HSCT). This study included 19 patients with sHVOD. Give intravenous infusion of defibrinated sodium, 5~60mg/kg per day, with a median use time of 15 days (2-61 days). The results showed that 42% of patients achieved complete remission (CR), and the overall survival (os) rate 100 days after transplantation reached 32%, and defibrinated sodium was well tolerated by patients.

Not only that, defibrotide sodium has shown positive therapeutic effects in multiple clinical trials, and it also has a certain effect in the treatment of multiple myeloma. Its launch meets the treatment needs of this rare and fatal disease.

Bruce Cozad, chairman and CEO of Jazz Pharmaceuticals, said: "(Defibrotide) is a clinically significant treatment advance because it represents a potentially life-saving intervention for patients with VOD who previously had no approved option for patients in the United States."