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Mohty, Mohamad; Blaise, Didier; Peffault de Latour, Régis; Labopin, Myriam; Bourhis, Jean Henri; Bruno, Benedicte; Ceballos, Patrice; Detrait, Marie; Gandemer, Virginie; Huynh, Anne; Izadifar-Legrand, Faezeh; Jubert, Charlotte; Labussière-Wallet, Hélène; Lebon, Delphine; Maury, Sébastien; Paillard, Catherine; Pochon, Cécile; Renard, Cecile; Rialland, Fanny; Schneider, Pascale; Sirvent, Anne; Asubonteng, Kobby; Guindeuil, Gwennaëlle; Yakoub-Agha, Ibrahim;

Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study

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  • Media type: E-Article
  • Title: Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study
  • Contributor: Mohty, Mohamad; Blaise, Didier; Peffault de Latour, Régis; Labopin, Myriam; Bourhis, Jean Henri; Bruno, Benedicte; Ceballos, Patrice; Detrait, Marie; Gandemer, Virginie; Huynh, Anne; Izadifar-Legrand, Faezeh; Jubert, Charlotte; Labussière-Wallet, Hélène; Lebon, Delphine; Maury, Sébastien; Paillard, Catherine; Pochon, Cécile; Renard, Cecile; Rialland, Fanny; Schneider, Pascale; Sirvent, Anne; Asubonteng, Kobby; Guindeuil, Gwennaëlle; Yakoub-Agha, Ibrahim;
  • Published: Springer Science and Business Media LLC, 2023
  • Published in: Bone Marrow Transplantation, 58 (2023) 4, Seite 367-376
  • Language: English
  • DOI: 10.1038/s41409-022-01900-6
  • ISSN: 0268-3369; 1476-5365
  • Origination:
  • Footnote:
  • Description: AbstractVeno-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.
  • Access State: Open Access