What is the effect of defibrotide?

What kind of drug is defibrinoside? How effective is it? Defibrotide is currently recognized as the most promising new drug for the treatment of hepatic veno-occlusive disease (HVOD) after hematopoietic stem cell transplantation (HSCT). Based on its anticoagulant, fibrinolysis-promoting, and anti-inflammatory effects, Richardson et al. first explored its application in the treatment of HVOD after HSCT. This study included 19 sHVOD patients. The dosage of defibrotide is 5~60mg/kg per day, intravenous infusion, and the median usage time is 15d (2-61d). The results showed that 42% of patients achieved complete remission (CR), and the overall survival (os) rate 100 days after transplantation reached 32%, and patients could tolerate defibrination well. Since then, multiple clinical trials have confirmed the effectiveness of defibrotide in the treatment of HVOD.

The recommended dose of defibrotide for adult and pediatric patients is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. The dose should be based on the patient's baseline weight, which is defined as the patient's weight before the HSCT preparation protocol. Defibrotide was administered for a minimum of 21 days. If signs and symptoms of hepatic VOD have not resolved after 21 days, continue defibrotide until resolution of VOD or up to a maximum of 60 days. Defibrotide must be diluted prior to infusion.

Prior to administration of defibrotide, confirm that the patient is not experiencing clinically significant bleeding and is hemodynamically stable on no more than one vasopressor. Defibrotide was administered by constant intravenous infusion over a 2-hour period. Administer the diluted defibrotide solution using an infusion set equipped with a 0.2 μm in-line filter. Flush the intravenous administration line (peripheral or central) with 5% dextrose injection, USP, or 0.9% sodium chloride injection, USP, before and immediately after administration.

Hypersensitivity reactions to defibrotide: including rash, urticaria and angioedema, occur in less than 2% of patients. Monitor patients for hypersensitivity reactions. If symptoms are severe, use should be discontinued, treated according to standard of care, and monitored until symptoms resolve. The most common adverse reactions (incidence ≥10%, no causal relationship) when taking defibrotide are hypotension, diarrhea, vomiting, nausea and epistaxis.