Off-label use of mycophenolate mofetil in the treatment of rare and complex rheumatic connective tissue diseases. - Université de Lille Accéder directement au contenu
Article Dans Une Revue Clinical and experimental rheumatology Année : 2022

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

M. Bandeira
  • Fonction : Auteur
A. Vieira
  • Fonction : Auteur
V. Guimarães
  • Fonction : Auteur
T. Bento
  • Fonction : Auteur
L. Beretta
  • Fonction : Auteur
A. Cere
  • Fonction : Auteur
G. Chehab
  • Fonction : Auteur
J. Milas-Ahić
  • Fonction : Auteur
U. Müller-Ladner
  • Fonction : Auteur
G. Nagy
  • Fonction : Auteur
Y. Piette
  • Fonction : Auteur
S. Rednic
  • Fonction : Auteur
M. Schneider
  • Fonction : Auteur
V. Smith
  • Fonction : Auteur
M. Cutolo
  • Fonction : Auteur
J. E. Fonseca
  • Fonction : Auteur
V. C. Romão
  • Fonction : Auteur

Résumé

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.

Dates et versions

hal-04503169 , version 1 (13-03-2024)

Identifiants

Citer

M. Bandeira, A. Vieira, V. Guimarães, T. Bento, Zahir Amoura, et al.. Off-label use of mycophenolate mofetil in the treatment of rare and complex rheumatic connective tissue diseases.. Clinical and experimental rheumatology, 2022, Clinical and experimental rheumatology, ⟨10.55563/clinexprheumatol/v1e7s2⟩. ⟨hal-04503169⟩
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