去纤苷治疗肝静脉闭塞症的效果怎么样?

One trial included children undergoing HPCT (hematopoietic progenitor cell transplantation) and those who received defibrin during hospital admission. Demographic data and clinical course information for these patients were extracted from their health records.

Patients who underwent HPCT during the study period received defibrotide to treat HVOD (hepatic veno-occlusive disease); nine were girls. Most patients underwent HPCT for hematologic malignancies (8/14) and received matched unrelated donor transplantation (8/14).

Conditioning regimen included total body irradiation with cyclophosphamide (5/14) and busulfan followed by cyclophosphamide (7/14). HVOD was diagnosed from transplant day 4 to +33 (median: +10.5); defibrotide was initiated from transplant day 4 to +40 (median: +12).

The median initial dose of defibrotide was 33 mg/kg/day (11-40 mg/kg/day); the median maximum dose of defibrotide was 38.5 mg/kg/day (11-81 mg/kg/day). The average duration of defibrotide treatment was 16 days (range 4-37 days).

Defibrotide was discontinued due to clinical improvement (9), death (3), drug unavailability (1), and neurotoxicity (1). During defibrotide treatment, gastrointestinal bleeding was observed in two patients and intracranial hemorrhage in one patient. The survival rate by day +100 was 79%.

Conclusion: Defibrotide appears to be an effective and relatively safe treatment for children with HVOD.

Common side effects of defibrotide include hypotension, diarrhea, vomiting, nausea, and nosebleeds. The symptoms of side effects vary depending on the patient's constitution and condition. It should be noted that concomitant use of defibrotide with antithrombotic or fibrinolytic drugs is contraindicated due to an increased risk of bleeding. It is recommended that patients take medication under the guidance of a doctor and receive symptomatic treatment.

Defibrinoside is the first FDA-approved drug to treat severe hepatic venule occlusion. It prevents the formation of blood clots and helps dissolve blood clots by increasing the levels of prostaglandin I2, E2 and prostacyclin, changing platelet activity, increasing tissue plasminogen activator function and reducing the activity of tissue plasminogen activator inhibitor.

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References

Bulley SR, Strahm B, Doyle J, Dupuis LL. Defibrotide for the treatment of hepatic veno-occlusive disease in children. Pediatr Blood Cancer. 2007 Jun 15;48(7):700-4. doi: 10.1002/pbc.20934. PMID: 16786586.