{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["58(4)"],"submitter":["Mohty M"],"funding":["Jazz Pharmaceuticals"],"pubmed_abstract":["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/m"],"journal":["Bone marrow transplantation"],"pagination":["367-376"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC10073014"],"repository":["biostudies-literature"],"pubmed_title":["Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study."],"pmcid":["PMC10073014"],"pubmed_authors":["Mohty M","Huynh A","Pochon C","Paillard C","Peffault de Latour R","Ceballos P","Asubonteng K","Izadifar-Legrand F","Dalle JH","Maury S","Yakoub-Agha I","Jubert C","Detrait M","Bruno B","Lebon D","Sirvent A","Bourhis JH","Schneider P","Labopin M","Labussiere-Wallet H","Renard C","Guindeuil G","Rialland F","Blaise D","Gandemer V"],"additional_accession":[]},"is_claimable":false,"name":"Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study.","description":"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/m","dates":{"release":"2023-01-01T00:00:00Z","publication":"2023 Apr","modification":"2025-04-22T13:05:41.302Z","creation":"2025-04-06T00:32:17.822Z"},"accession":"S-EPMC10073014","cross_references":{"pubmed":["36564486"],"doi":["10.1038/s41409-022-01900-6"]}}