去纤苷的治疗效果怎么样

In the EU, defibrotide is approved for the treatment of severe veno-occlusive disease/sinus obstructive syndrome (VOD/SOS) after hematopoietic cell transplantation in patients older than 1 month, and in the United States, defibrotide is approved for the treatment of VOD/SOS after hematopoietic cell transplantation for renal/pulmonary dysfunction. A meta-analysis of trials used published literature to estimate the incidence and risk of VOD/SOS after intravenous deficinitin prophylaxis.

Methods: PubMed, Embase and Web of Science were searched for defibrinoside studies in VOD/SOS "Prevention" or "Prevention", excluding phase I studies, case reports, studies with less than 10 patients, and reviews.

Results: 733 records were retrieved; 24 cases met the inclusion criteria, of which 20 cases (N=3005) evaluated intravenous infusion of defibrotide for the prevention of VOD/SOS.静脉注射去纤苷的VOD/SOS总发生率为5%，成人和儿科患者的发生率分别为5%和8%。

Data from 8 studies comparing intravenous prophylaxis with a control group (eg, heparin, no prophylaxis) showed that the incidence of VOD/SOS in the control group was 16%. Compared with the control group, the hazard ratio for VOD/SOS prophylaxis with defibrotide was 0.30.

CONCLUSIONS: This analysis demonstrates that the incidence of VOD/SOS after intravenous defibrotide prophylaxis is lower regardless of age group and that in a patient population at high risk for VOD/SOS, the relative risk of VOD/SOS is lower with intravenous defibrotide prophylaxis compared with controls.

Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening conditioning complication during hematopoietic stem cell transplantation (HSCT) or HSCT-free chemotherapy, with a historically reported average incidence of 13.7% after HSCT. Typical symptoms of VOD/SOS may include hyperbilirubinemia, painful hepatomegaly, weight gain, and ascites.

In a phase 3, historically controlled, multicenter trial of adults and children with VOD/SOS and MOD/MOF (defibrotide: n=102; control group treated before defibrotide became available: n=32), defibrotide resulted in a significant improvement in survival at day +100 after HSCT (38.2%) compared with the control group (25.0%; propensity analysis estimated between-group difference: 23%; P=0.0109). The most common AEs were hypotension and diarrhea; the incidence of common hemorrhagic AEs was similar in the defibrotide group and the historical control group (64% and 75%, respectively).

Defibrotide is effective in the treatment of hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), and adult and pediatric patients with renal or pulmonary dysfunction after hematopoietic stem cell transplantation (HSCT). It is recommended that patients take medication under the guidance of a doctor and receive symptomatic treatment.

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References

[1.]Corbacioglu S, Topaloglu O, Aggarwal S. A Systematic Review and Meta-Analysis of Studies of Defibrotide Prophylaxis for Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome. Clin Drug Investig. 2022 Jun;42(6):465-476. doi: 10.1007/s40261-022-01140-y. Epub 2022 May 20. PMID: 35594010; PMCID: PMC9188533.

[2.]Richardson PG, Carreras E, Iacobelli M, Nejadnik B. The use of defibrotide in blood and marrow transplantation. Blood Adv. 2018 Jun 26;2(12):1495-1509. doi: 10.1182/bloodadvances.2017008375. Erratum in: Blood Adv. 2018 Aug 14;2(15):1853. PMID: 29945939; PMCID: PMC6020812.