Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS)

Richardson, Paul and Aggarwal, Saurabh and Topaloglu, Ozlem and Villa, Kathleen F. and Corbacioglu, Selim (2019) Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS). BONE MARROW TRANSPLANTATION, 54 (12). pp. 1951-1962. ISSN 0268-3369, 1476-5365

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Abstract

Veno-occlusive disease (VOD), also called sinusoidal obstruction syndrome (SOS), is a potentially life-threatening complication of hematopoietic stem cell transplantation (HSCT) conditioning or high-dose nontransplant chemotherapy. VOD/SOS with multi-organ dysfunction (MOD) is associated with a mortality rate of >80%. Defibrotide (25 mg/kg/day) is approved to treat hepatic VOD/SOS with renal or pulmonary dysfunction post HSCT in the United States and to treat severe hepatic VOD/SOS in patients > 1 month of age in the European Union. A random effects model was used for pooling data from 17 systematically chosen defibrotide studies. For patients in these reports (n = 2598), and those in the subset of 10 reports of patients treated with similar to 25 mg/kg/day (n = 1691), estimated Day + 100 survival rates were 54% and 56%, respectively. Among those patients treated with similar to 25 mg/kg/day, estimated Day + 100 survival was 44% among patients with MOD and 71% in patients without MOD; survival was 41% and 70%, respectively, for the population of patients receiving any dose of defibrotide. Safety results were not pooled owing to differences in reporting methodology but were generally consistent with the known tolerability profile of defibrotide. This analysis provides the largest assessment of survival in patients treated with defibrotide for VOD/SOS with or without MOD.

Item Type: Article
Uncontrolled Keywords: STEM-CELL TRANSPLANTATION; MARROW-TRANSPLANTATION; OCCLUSIVE DISEASE; EUROPEAN-SOCIETY; SIGNIFICANT TOXICITY; SEVERITY CRITERIA; SINGLE-CENTER; RISK-FACTORS; BLOOD; CHILDREN;
Subjects: 600 Technology > 610 Medical sciences Medicine
Divisions: Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation
Depositing User: Dr. Gernot Deinzer
Date Deposited: 23 Mar 2020 08:00
Last Modified: 23 Mar 2020 08:00
URI: https://pred.uni-regensburg.de/id/eprint/25754

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