Titre :

Off-label use of mycophenolate mofetil in the treatment of rare and complex rheumatic connective tissue diseases.

Auteur(s) :

Bandeira, M. [Auteur]
Vieira, A. [Auteur]
Guimarães, V. [Auteur]
Bento, T. [Auteur]
Amoura, Zahir [Auteur]
CHU Pitié-Salpêtrière [AP-HP]
Arnaud, Laurent [Auteur]
Les Hôpitaux Universitaires de Strasbourg [HUS]
Beretta, L. [Auteur]
Cere, A. [Auteur]
Chehab, G. [Auteur]
Hachulla, Eric [Auteur]
Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286
Milas-Ahić, J. [Auteur]
Müller-Ladner, U. [Auteur]
Nagy, G. [Auteur]
Piette, Y. [Auteur]
Rednic, S. [Auteur]
Schneider, M. [Auteur]
Smith, V. [Auteur]
Cutolo, M. [Auteur]
Fonseca, J. E. [Auteur]
Romão, V. C. [Auteur]

Titre de la revue :

Clinical and experimental rheumatology

Nom court de la revue :

Clin Exp Rheumatol

Date de publication :

2022-03-31

ISSN :

0392-856X

Mot(s)-clé(s) en anglais :

mycophenolate mofetil
connective tissue diseases
rheumatology

Discipline(s) HAL :

Sciences du Vivant [q-bio]

Résumé en anglais : [en]

Objectives: We aimed to investigate the clinical off-label use of mycophenolate mofetil (MMF), including its safety and efficacy in patients with rare and complex rheumatic connective tissue diseases (rCTDs). Methods: ...
Lire la suite >Objectives: We aimed to investigate the clinical off-label use of mycophenolate mofetil (MMF), including its safety and efficacy in patients with rare and complex rheumatic connective tissue diseases (rCTDs). Methods: A survey was distributed across experts from ERN-ReCONNET reference centres in order to assess the experience with MMF off-label use. Patient-level data of patients with rCTDs under treatment with MMF was also collected for analysis of safety and efficacy. Results: Twelve experts from eleven centres distributed throughout Europe (7 countries) answered the survey. The experience was concordant in that, despite of its off-label use, experts reported opting frequently for this therapeutic alternative with robust confidence on its efficacy and safety. The analysis of 108 patients with rCTDs under MMF revealed a good safety profile, as well as good clinical outcomes, especially for systemic lupus erythematosus and idiopathic inflammatory myopathies. The presence of interstitial lung disease was, as expected, associated with a worse clinical outcome despite use of MMF. Conclusions: MMF is widely used in reference centres for rCTDs. Its safety profile and efficacy seem to be recognised by experts and demonstrated with patient-level analysis. While selected rCTDs will likely remain an off-label indication for MMF, robust data seem to support this therapy as an appropriate alternative for safely and effectively treating many manifestations of rCTDs.Lire moins >

Langue :

Anglais

Audience :

Internationale

Vulgarisation :

Non

Établissement(s) :

Université de Lille
Inserm
CHU Lille

Collections :

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

Date de dépôt :

2024-01-12T01:57:42Z
2024-03-13T14:22:42Z