Off-label use of mycophenolate mofetil in the treatment of rare and complex rheumatic connective tissue diseases.
Clin Exp Rheumatol
; 40 Suppl 134(5): 32-39, 2022 May.
Article
em En
| MEDLINE
| ID: mdl-35349418
ABSTRACT
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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Doenças Reumáticas
/
Doenças do Tecido Conjuntivo
/
Lúpus Eritematoso Sistêmico
Tipo de estudo:
Diagnostic_studies
Limite:
Humans
Idioma:
En
Revista:
Clin Exp Rheumatol
Ano de publicação:
2022
Tipo de documento:
Article