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Hepatic veno-occlusive disease (HVOD) is one of the serious complications after hematopoietic stem cell transplantation (HSCT), and the mortality rate in patients with severe HVOD can be as high as 100%. Defibrotide is a mixture of single-stranded oligonucleotides with anti-thrombotic and fibrinolytic effects. In recent years, multiple clinical research results have shown that defibrotide is a safe and effective drug for preventing and treating HVOD after HSCT.

Defibrotide is an adenosine receptor agonist with multiple effects. The adenosine A1/A2 receptors of endothelial cells are involved in the regulation of endothelial cells and the response of endothelial cells to injury. Defibrotide can act on these receptors to produce a variety of downstream effects. Defibrotide can also reduce the expression of cell adhesion molecules on the surface of endothelial cells, thereby reducing the adhesion of leukocytes to vascular endothelial cells and reducing the inflammatory damage of endothelial cells. In addition, defibrotide can also promote the release of prostaglandin I2 (PGI2) and prostaglandin E2 (PGE2), thereby causing blood vessel dilation, inhibiting platelet aggregation, and reducing ischemic damage. Studies have shown that defibrotide can significantly increase the expression of thrombin regulatory protein (TM) and tissue factor pathway inhibitors to produce anticoagulant effects.

The recommended dose of Defibrotide is 6.25 mg/kg given every 6 hours as a 2-hour intravenous infusion. Treatment lasts for a minimum of 21 days. If signs and symptoms of VOD do not resolve after 21 days, continue treatment until resolution.

Defibrotide has received marketing authorization from the European Commission for the treatment of severe hepatic VOD in hematopoietic stem cell transplantation (HSCT). A phase 3 randomized controlled trial of defibrination to prevent hepatic VOD in pediatric transplant patients has also been completed. Defibrotide has been generally well tolerated; the most common side effects observed during premarketing use to treat hepatic VOD were bleeding, hypotension, and coagulopathy.

If you experience side effects while taking defibrinoside, please report it to your attending doctor immediately and handle it in accordance with the doctor's instructions. Do not use the medicine without authorization to avoid unnecessary drug reactions.