Currently Enrolling Trials
Defitelio (defibrotide sodium) is the sodium salt of a mixture of single-stranded oligodeoxyribonucleotides derived from porcine mucosal DNA, which has been shown to have antithrombotic, anti-inflammatory and anti-ischemic properties.
Defitelio is specifically indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).
Defitelio is supplied as a solution for intravenous infusion. The recommended dose of Defitelio is 6.25 mg/kg every 6 hours given as a 2-hour intravenous infusion. Treat for a minimum of 21 days. If after 21 days signs and symptoms of VOD have not resolved, continue treatment until resolution.
The FDA approval of Defitelio was based on three clinical studies in patients with hepatic VOD with renal or pulmonary dysfunction following HSCT who were treated with Defitelio at the recommended 6.25 mg/kg every 6 hours. The efficacy of Defitelio was based on survival at Day + 100 after HSCT. In Study 1, the survival rate was 38% at 100 days after transplantation. In Study 2 the survival rate was 44% at 100 days after transplantation. In Study 3, the Day + 100 survival was 45%. Based on published reports and analyses of patient level data for individuals with hepatic VOD with renal or pulmonary dysfunction who received supportive care or interventions other than Defitelio, the expected Day +100 survival rates are 21% to 31%.
Adverse effects associated with the use of Defitelio may include, but are not limited to, the following:
Mechanism of Action
Defitelio (defibrotide sodium) enhances the enzymatic activity of plasmin to hydrolyze fibrin clots in vitro. The exact mechanism of action of defibrotide is not fully understood.
For additional information regarding Defitelio or hepatic VOD, please visit https://www.jazzpharma.com