去纤苷治疗肝小静脉闭塞病的效果？

Author: Medicalhalo

Release time: 2025-10-19 11:44:20

Defibrotide has a better effect in treating hepatic veno-occlusive disease, and patients can take medication under the guidance of a doctor.

Defibrotide was first developed by Gentium Pharmaceuticals in Italy and was approved for marketing in the EU in October 2013. After Gentium Pharmaceuticals was acquired by American Jazz Pharmaceuticals, defibrotide was approved by the U.S. Food and Drug Administration (FDA) for marketing in the United States on March 30, 2016. It is the first drug approved by the U.S. FDA for the treatment of severe hepatic veno-occlusive disease.

hepatic veno-occlusive disease

Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is the most common complication in early patients after hematopoietic stem cell transplantation (HSCT). In severe cases, VOD/SOS can be accompanied by multiple organ failure, and the mortality rate exceeds 80% within +100 days. Defibrotide has been proposed for the treatment of SOS due to its ability to restore the thrombo-fibrinolytic balance and protect endothelial cells.

Efficacy

Defibrotide is a polydisperse oligonucleotide obtained from porcine intestinal mucosa and prepared by controlled depolymerization of DNA. It is a nucleic acid polymer, primarily single-stranded, with anti-ischemic and anti-thrombotic properties.

The efficacy and safety of defibrotide in the treatment of veno-occlusive disease (VOD) occurring after high-dose chemotherapy and hematopoietic stem cell transplantation have now been well established in Phase II-III trials. A recent randomized phase III trial in pediatric patients also demonstrated its role in preventing VOD.

Preclinical studies have reported the inhibitory effects of defibrotide on myeloma cell growth through anti-angiogenic effects and modulation of tumor-microenvironment interactions. A recent phase II trial highlighted the efficacy and safety of the defibrotide-thalidomide-melphalan combination in relapsed/refractory multiple myeloma.

Defibrotide is effective in the prevention and treatment of veno-occlusive disease. Recent experimental results suggest that defibrotide may belong to a new generation of anticancer drugs capable of blocking tumor angiogenesis. In multiple myeloma, defibrotide can overcome the prothrombotic effects of thalidomide on endothelial cells.

Defibrotide in the treatment of hepatic veno-occlusive disease

Defibrotide was recently approved in the EU for the treatment of severe hepatic veno-occlusive disease (VOD), also known as sinus tract obstruction syndrome, during hematopoietic stem cell transplantation (HSCT) therapy. It is suitable for adults, teenagers, children and babies over 1 month old.

Defibrotide is also available in the United States through an expanded regimen. Defibrotide is thought to protect endothelial cells and restore the thrombo-fibrinolytic balance in VOD. In a multicenter, phase III trial, patients with severe hepatic VOD and multiorgan failure who received intravenous defibrotide 6.25 mg/kg every 6 hours after HSCT had a significantly higher complete response rate (primary endpoint) at day +100 and a significantly lower mortality at day +100 compared with a historical control group.

The efficacy of defibrotide in severe hepatic VOD after HSCT is also supported by results from phase II dose-finding studies, compassionate use data and information from independent transplant registries. Intravenous defibrotide is generally well tolerated in patients with severe hepatic VOD after HSCT and is not associated with an increased risk of bleeding adverse events.

In conclusion, defibrotide is the only drug approved (in the EU) for severe hepatic VOD after HSCT and represents a useful advance in the treatment of this disease.

Defibrinoside

1. Recommended dosage:

It is recommended that adult and pediatric patients receive 6.25 mg/kg as an intravenous infusion over 2 hours, once every 6 hours. This dose should be based on the patient's basal body weight and determined prior to preparation for HSCT (HSCs transplantation).

2. Administration time:

The administration period of defibrotide was 21 days. If symptoms of hepatic venule obstruction appear after 21 days, antifibrogenic drug treatment needs to be continued until the end of the treatment or extended to 60 days.

Summary

Defibrotide has good curative effect on hepatic veno-occlusive disease. The specific usage and dosage should be strictly in accordance with the doctor's instructions. Patients are not allowed to take the medicine privately to avoid improper use of medicines that may harm their health.