Title :

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

Author(s) :

Mohty, Mohamad [Auteur]
Centre de Recherche Saint-Antoine [CRSA]
Sorbonne Université [SU]
CHU Saint-Antoine [AP-HP]
Blaise, Didier [Auteur]
Institut Paoli-Calmettes [IPC]
Aix Marseille Université [AMU]
Peffault De Latour, Régis [Auteur]
Hopital Saint-Louis [AP-HP] [AP-HP]
Labopin, Myriam [Auteur]
Centre de Recherche Saint-Antoine [CRSA]
Sorbonne Université [SU]
CHU Saint-Antoine [AP-HP]
Bourhis, J. H. [Auteur]
Institut Gustave Roussy [IGR]
Bruno, Benedicte [Auteur]
Hôpital Jeanne de Flandre [Lille]
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Ceballos, P. [Auteur]
Centre Hospitalier Régional Universitaire [Montpellier] [CHRU Montpellier]
Detrait, Marie [Auteur]
Service d'Hématologie [CHRU Nancy]
Gandemer, V. [Auteur]
Centre Hospitalier Universitaire [Rennes]
Huynh, A. [Auteur]
Service Hématologie - IUCT-Oncopole [CHU Toulouse]
Izadifar-Legrand, F. [Auteur]
Institut Paoli-Calmettes [IPC]
Jubert, C. [Auteur]
CHU Bordeaux
Labussière-Wallet, H. [Auteur]
Service d’Hématologie [Centre Hospitalier Lyon Sud - HCL]
Lebon, D. [Auteur]
CHU Amiens-Picardie
Maury, S. [Auteur]
Hôpital Henri Mondor
Paillard, C. [Auteur]
Hôpital de Hautepierre [Strasbourg]
Pochon, C. [Auteur]
Renard, Cécile [Auteur]
Institut d'hématologie et d'oncologie pédiatrique [CHU - HCL] [IHOPe]
Rialland, F. [Auteur]
Centre Hospitalier Universitaire de Nantes = Nantes University Hospital [CHU Nantes]
Schneider, P. [Auteur]
CHU Rouen
Sirvent, A. [Auteur]
Centre Hospitalier Régional Universitaire [Montpellier] [CHRU Montpellier]
Asubonteng, K. [Auteur]
Guindeuil, Gwennaëlle [Auteur]
Jazz Pharmaceuticals [Lyon]
Yakoub-Agha, Ibrahim [Auteur]
Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286
Dalle, J. H. [Auteur]
AP-HP Hôpital universitaire Robert-Debré [Paris]

Journal title :

Bone Marrow Transplantation

Abbreviated title :

Bone Marrow Transplant

Publication date :

2022-12-25

ISSN :

1476-5365

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

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 ...
Show more >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.Show less >

Language :

Anglais

Peer reviewed article :

Oui

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Submission date :

2024-01-11T23:49:43Z
2024-03-08T15:06:59Z