Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study.
Fiche publication
Date publication
décembre 2022
Journal
Bone marrow transplantation
Auteurs
Membres identifiés du Cancéropôle Est :
Dr POCHON Cécile
Tous les auteurs :
Mohty M, Blaise D, Peffault de Latour R, Labopin M, Bourhis JH, Bruno B, Ceballos P, Detrait M, Gandemer V, Huynh A, Izadifar-Legrand F, Jubert C, Labussière-Wallet H, Lebon D, Maury S, Paillard C, Pochon C, Renard C, Rialland F, Schneider P, Sirvent A, Asubonteng K, Guindeuil G, Yakoub-Agha I, Dalle JH
Lien Pubmed
Résumé
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.
Référence
Bone Marrow Transplant. 2022 12 23;: