biostudies-literatureUnknown58(4)Mohty MJazz PharmaceuticalsVeno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of haematopoietic cell transplantation (HCT) conditioning. The DEFIFrance post-marketing registry study evaluated effectiveness and safety in patients who received defibrotide. It collected retrospective/prospective patient data from 53 French HCT centres from July 2014 to March 2020. Primary endpoints were survival and complete response (CR; total serum bilirubin <2 mg/dL, multiorgan failure resolution) at Day 100 post-HCT among patients with severe/very severe VOD/SOS. A secondary endpoint was evaluation of treatment-emergent serious adverse events (TESAEs) of interest. Of 798 patients analysed, 251 and 81 received defibrotide treatment for severe/very severe VOD/SOS and mild/mBone marrow transplantation367-376https://www.ebi.ac.uk/biostudies/studies/S-EPMC10073014biostudies-literatureReal-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study.PMC10073014Mohty MHuynh APochon CPaillard CPeffault de Latour RCeballos PAsubonteng KIzadifar-Legrand FDalle JHMaury SYakoub-Agha IJubert CDetrait MBruno BLebon DSirvent ABourhis JHSchneider PLabopin MLabussiere-Wallet HRenard CGuindeuil GRialland FBlaise DGandemer VfalseReal-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study.Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of haematopoietic cell transplantation (HCT) conditioning. The DEFIFrance post-marketing registry study evaluated effectiveness and safety in patients who received defibrotide. It collected retrospective/prospective patient data from 53 French HCT centres from July 2014 to March 2020. Primary endpoints were survival and complete response (CR; total serum bilirubin <2 mg/dL, multiorgan failure resolution) at Day 100 post-HCT among patients with severe/very severe VOD/SOS. A secondary endpoint was evaluation of treatment-emergent serious adverse events (TESAEs) of interest. Of 798 patients analysed, 251 and 81 received defibrotide treatment for severe/very severe VOD/SOS and mild/m2023-01-01T00:00:00Z2023 Apr2025-04-22T13:05:41.302Z2025-04-06T00:32:17.822ZS-EPMC100730143656448610.1038/s41409-022-01900-6