Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study - Archive ouverte HAL Accéder directement au contenu
Article Dans Une Revue Bone Marrow Transplantation Année : 2022

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

(1, 2) , (3) , (4) , (5) , (6) , (7) , (8) , (9) , (10, 11) , (12) , , (13) , , (14, 15) , (16, 17) , (18) , (19) , , , (20, 21) , , , , ,
1
2
3
4
5
6
7
8
9
10
11
12
13
14
15
16
17
18
19
20
21
Faezeh Izadifar-Legrand
  • Fonction : Auteur
Hélène Labussière-Wallet
  • Fonction : Auteur
Cecile Renard
  • Fonction : Auteur
Fanny Rialland
Anne Sirvent
  • Fonction : Auteur
Kobby Asubonteng
  • Fonction : Auteur
Gwennaëlle Guindeuil
  • Fonction : Auteur
Ibrahim Yakoub-Agha
Jean-Hugues Dalle
  • Fonction : Auteur

Résumé

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/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.
Fichier principal
Vignette du fichier
s41409-022-01900-6.pdf (913.52 Ko) Télécharger le fichier
Origine : Publication financée par une institution

Dates et versions

hal-03915550 , version 1 (23-01-2023)

Identifiants

Citer

Mohamad Mohty, Didier Blaise, Régis Peffault de Latour, Myriam Labopin, Jean Henri Bourhis, et al.. Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study. Bone Marrow Transplantation, 2022, ⟨10.1038/s41409-022-01900-6⟩. ⟨hal-03915550⟩
0 Consultations
0 Téléchargements

Altmetric

Partager

Gmail Facebook Twitter LinkedIn More