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去纤苷的功效与作用及注意事项?

Author: Medicalhalo
Release time: 2025-10-19 11:44:20
The efficacy and effects of defibrinoside mainly include promoting fibrinolysis, improving microcirculation, reducing mortality, reducing the need for liver transplantation, etc. The precautions for defibrinoside mainly include bleeding, allergic reactions, etc.

Indications for defibrotide

It is approved for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD) (also known as sinusoidal obstruction syndrome (SOS)) associated with renal or pulmonary dysfunction after hematopoietic stem cell transplantation (HSCT).

Mechanism of action of defibrotide

Defibrotide has multiple complex mechanisms of action, including anti-inflammatory, anti-atherosclerotic, anti-ischemic and anti-thrombotic properties. Research on defibrinoside also shows that increased plasmin activity promotes fibrinolysis by upregulating tissue plasminogen activator and inhibiting tissue factor pathways, reduces circulating levels of plasminogen activator inhibitor-1, and increases endogenous prostaglandin concentrations (I2 and E2), regulating thrombomodulin, platelets, and fibrinolysis.

Effects and functions of defibrinoside

1. Promote fibrinolysis: Defibrinoside can enhance the hydrolase activity of plasmin on fibrin clots in vitro and help prevent thrombosis.

2. Improve microcirculation: By regulating the function of endothelial cells, defibrotide can increase the release of post-inflammatory factors and promote the activation of plasminogen, thereby improving liver microcirculation and metabolism.

3. Reduce mortality: In the treatment of hepatic veno-occlusive disease (HVOD), defibrotide has been proven to significantly reduce mortality.

4. Reduce the need for liver transplantation: The use of defibrotide reduces the need for liver transplantation due to HVOD.

Efficacy of defibrotide

In a clinical study, 102 patients with clinical diagnosis of VOD on day 21 after transplantation and renal or pulmonary failure on day 28 after transplantation were included, as well as 32 historical control patients who met the diagnostic criteria for VOD combined with multiple organ failure.

Patients received defibrotide 25 mg/kg/day as a 2-hour infusion in 4 doses for at least 21 days. When compared to historical control patients, the median duration of defibrotide treatment was 21.5 days.

The 100-day survival rate after HSCT was 38.2% in the defibrotide group and 25% in the historical control group, with an absolute treatment difference of 13.2%. The complete response rate was 25.5% in the defibrotide-treated group and 12.5% ​​in the historical control group, for an absolute treatment difference of 13%.

The survival rate on day 180 after HSCT was 32.4% in the defibrotide group and 25% in the historical control group, with a treatment difference of 16.4%. The complete remission rate in children treated with defibrotide was 36%, compared with 7% in the historical control group. The survival rate 100 days after transplantation was 34% for patients who received dialysis, compared with 9% for patients who did not receive dialysis.

Precautions for defibrinoside

1. Bleeding: Patients with active bleeding should not start using defibrinoside. The drug will increase the activity of plasmin and may increase the risk of bleeding in patients with VOD after HSCT. If bleeding occurs, treatment should be discontinued, the underlying cause treated, and supportive care provided until bleeding stops.

2. Allergic reactions: There have been reports of allergic reactions to defibrinoside, such as rash, urticaria, angioedema, and allergic reactions. Patients should be monitored for allergic reactions during medication, especially those with a history of previous exposure. If severe allergic reaction occurs discontinue medication and treat according to standard of care.

3. Pregnancy medication: The efficacy and safety of defibrotide during human pregnancy are not yet clear, but animal models have shown that there is a potential risk of reduced implantation rate and miscarriage, and it should be banned or used with caution during pregnancy.

4. Breastfeeding precautions: The safety of defibrinoside in breastfeeding has not been determined, and there is no information on the presence of defibrinoside in human milk or its effects on milk production or breastfed infants. Breastfeeding is not recommended because breastfed babies may bleed.

Summary

Defibrotide should be used under the guidance of a doctor because there may be some side effects, such as an increased risk of bleeding. During treatment with defibrotide, patients should have regular blood tests to assess treatment effectiveness and monitor potential side effects.

References:

Baker DE, Demaris K. Defibrotide. Hosp Pharm. 2016 Nov;51(10):847-854. doi: 10.1310/hpj5110-847. PMID: 27928191; PMCID: PMC5135434.

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