%0 Journal Article %T Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study %+ CHU Saint-Antoine [AP-HP] %+ Centre de Recherche Saint-Antoine (CRSA) %+ Centre de Recherche en Cancérologie de Marseille (CRCM) %+ Institut Paoli-Calmettes (IPC) %+ Département d'hématologie [Gustave Roussy] %+ Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille) %+ Hôpital Lapeyronie [Montpellier] (CHU) %+ Service d'Hématologie [CHRU Nancy] %+ Institut de Génétique et Développement de Rennes (IGDR) %+ Institut Universitaire du Cancer de Toulouse - Oncopole (IUCT Oncopole - UMR 1037) %+ Groupe hospitalier Pellegrin %+ Centre Hospitalier Lyon Sud [CHU - HCL] (CHLS) %+ CHU Amiens-Picardie %+ HEMATIM - Hématopoïèse et immunologie - UR UPJV 4666 (HEMATIM) %+ Université Paris-Est Créteil Val-de-Marne - Faculté de médecine (UPEC Médecine) %+ Hôpital Henri Mondor %+ Centre d'Investigation Clinique [CHU Clermont-Ferrand] (CIC 1405) %+ Service d'Oncologie Pédiatrique [CHRU Nancy] %+ Service d’Hématologie [Centre Hospitalier Lyon Sud - HCL] %+ Centre hospitalier universitaire de Nantes (CHU Nantes) %+ Service de pédiatrie médicale et médecine de l'adolescent [Rouen] %+ Physiopathologie, Autoimmunité, maladies Neuromusculaires et THErapies Régénératrices (PANTHER) %+ CHU Montpellier %+ Jazz Pharmaceuticals, Inc %+ Jazz Pharmaceuticals [Lyon] %+ Institute for Translational Research in Inflammation - U 1286 (INFINITE (Ex-Liric)) %+ Hôpital Robert Debré Paris %A Mohty, Mohamad %A Blaise, Didier %A Peffault de Latour, Régis %A Labopin, Myriam %A Bourhis, Jean Henri %A Bruno, Benedicte %A Ceballos, Patrice %A Detrait, Marie %A Gandemer, Virginie %A Huynh, Anne %A Izadifar-Legrand, Faezeh %A Jubert, Charlotte %A Labussière-Wallet, Hélène %A Lebon, Delphine %A Maury, Sébastien %A Paillard, Catherine %A Pochon, Cécile %A Renard, Cecile %A Rialland, Fanny %A Schneider, Pascale %A Sirvent, Anne %A Asubonteng, Kobby %A Guindeuil, Gwennaëlle %A Yakoub-Agha, Ibrahim %A Dalle, Jean-Hugues %Z Jazz Pharmaceuticals %< avec comité de lecture %@ 0268-3369 %J Bone Marrow Transplantation %I Nature Publishing Group %8 2022-12-23 %D 2022 %R 10.1038/s41409-022-01900-6 %M 36564486 %K Drug therapy %K Haematopoietic stem cells %Z Life Sciences [q-bio]/Human health and pathologyJournal articles %X 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/moderate VOD/SOS post-HCT, respectively; 381 received defibrotide for VOD/SOS prophylaxis. In patients with severe/very severe VOD/SOS post-HCT, Kaplan-Meier-estimated CR at Day 100 was 74% (95% confidence interval [CI]: 66%, 81%). At Day 100, 137/251 (55%) were alive and in CR. Kaplan-Meier-estimated Day 100 post-HCT survival was 61% (95% CI: 55%, 67%) in patients with severe/very severe VOD/SOS. TESAEs of interest occurred in 29% of these patients; VOD/SOS-related mortality at 12 months was 15%. DEFIFrance represents the largest collection of real-world data on post-registration defibrotide use, supporting the real-world utility of defibrotide for patients with severe/very severe VOD/SOS post-HCT. %G English %2 https://u-picardie.hal.science/hal-03915550/document %2 https://u-picardie.hal.science/hal-03915550/file/s41409-022-01900-6.pdf %L hal-03915550 %U https://u-picardie.hal.science/hal-03915550 %~ HCL %~ UNIV-TLSE3 %~ UNIV-RENNES1 %~ CNRS %~ UNIV-AMU %~ UNIV-PICARDIE %~ APHP %~ CHLS %~ FNCLCC %~ IGR %~ PAOLI_CALMETTES %~ UPEC %~ U938 %~ IGDR %~ STATS-UR1 %~ IGDR-EGO %~ COMUE-NORMANDIE %~ UR1-UFR-SVE %~ UR1-HAL %~ UR1-SDV %~ TEST-UR-CSS %~ UNIV-RENNES %~ UNIROUEN %~ UNIV-LILLE %~ U1234PANTHER %~ SORBONNE-UNIVERSITE %~ SORBONNE-UNIV %~ CRCM %~ SU-MEDECINE %~ SU-MED %~ CHU-CLERMONTFERRAND %~ SU-TI %~ UR1-BIO-SA %~ U-PICARDIE %~ HEMATIM %~ ALLIANCE-SU %~ BGC %~ UNIV-UT3 %~ UT3-INP %~ UT3-TOULOUSEINP