Title :

Azacitidine for patients with Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic syndrome (VEXAS) and myelodysplastic syndrome: data from the French VEXAS registry

Author(s) :

Comont, Thibault [Auteur]
Université Toulouse III Paul Sabatier - Faculté de médecine Purpan [UTPS]
Pôle IUCT [CHU Toulouse]
Heiblig, Mael [Auteur]
Centre Hospitalier Lyon Sud [CHU - HCL] [CHLS]
Riviere, Etienne [Auteur]
Hôpital Haut-Lévêque [CHU Bordeaux]
Terriou, Louis [Auteur]
Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286
Rossignol, Julien [Auteur]
Hôpital Necker - Enfants Malades [AP-HP]
Centre de référence des mastocytoses [CEREMAST]
Bouscary, Didier [Auteur]
Hôpital Cochin [AP-HP]
Rieu, Virginie [Auteur]
Service de Médecine Interne [CHU Clermont-Ferrand]
Le Guenno, G. [Auteur]
Mathian, A. [Auteur]
Aouba, Achille [Auteur]
Université de Caen Normandie [UNICAEN]
CHU Caen
Vinit, Julien [Auteur]
Centre Hospitalier Chalon-sur-Saône William Morey
Dion, J. [Auteur]
Kosmider, O. [Auteur]
Terrier, B. [Auteur]
Georgin-Lavialle, Sophie [Auteur]
CHU Tenon [AP-HP]
Fenaux, P. [Auteur]
Mekinian, Arsène [Auteur]
CHU Saint-Antoine [AP-HP]

Journal title :

British Journal of Haematology

Abbreviated title :

Br. J. Haematol.

Volume number :

-

Pages :

-

Publication date :

2021-10-28

ISSN :

0007-1048

English keyword(s) :

myelodysplastic syndrome
VEXAS syndrome
azacitidine

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

Azacitidine can be effective in myelodysplastic syndromes (MDS) associated with inflammatory/autoimmune diseases. Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic syndrome (VEXAS) is a new monogenic autoinflammatory ...
Show more >Azacitidine can be effective in myelodysplastic syndromes (MDS) associated with inflammatory/autoimmune diseases. Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic syndrome (VEXAS) is a new monogenic autoinflammatory syndrome caused by somatic ubiquitin-like modifier-activating enzyme 1 (UBA1) mutation, often associated with MDS, whose treatment is difficult and not yet codified. Based on a French nationwide registry of 116 patients with VEXAS, we report the efficacy and safety of azacitidine treatment in 11 patients with VEXAS with MDS. Clinical response of VEXAS to azacitidine was achieved in five patients (46%), during 6, 8+, 12, 21, 27+ months respectively, suggesting that azacitidine can be effective in selected patients with VEXAS and associated MDS.Show less >

Language :

Anglais

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 :

2024-01-12T05:51:57Z
2024-02-28T08:38:47Z