Methotrexate treatment of rheumatological diseases off-label drug use Chinese expert consensus

Title: Methotrexate treatment of rheumatological diseases off-label drug use Chinese expert consensus
Edition: Original
Classification: Experts consensus
Field: Diagnosis and Treatment
Countries and regions: China
Guidelines users: Clinicians at all levels (rheumatology, pharmacy, pediatric rheumatology and other related fields)
Evidence classification method: The rating system of the Centre for Evidence-Based Medicine of the University of Oxford 2009 was adopted, in which the level of evidence was divided into level 1 (1a, 1b, 1c), level 2 (2a, 2b, 2c), level 3 (3a, 3b), level 4 and level 5, and the recommended level was divided into A, B, C, D and E
Development unit: Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University
Registration time: 2024-02-07
Registration number: PREPARE-2024CN258
Purpose of the guideline: Methotrexate (MTX) is an antimetabolic antitumor agent that inhibits DNA synthesis by inhibiting dihydrofolate reductase. Domestic MTX specification approved for the treatment of leukemia, head and neck cancer, lung cancer, breast cancer and other diseases. However, MTX is the first-line "anchor drug" in the treatment of rheumatoid arthritis in the field of rheumatology, and has obvious effects on systemic lupus erythematosus, dermatomyositis/myositis, vasculitis (such as anti-neutrophil cytoplasmic antibody associated vasculitis, giant cell arteritis), psoriatic arthritis, juvenile idiopathic arthritis and other rheumatological diseases, and has been written into domestic and foreign treatment guidelines. At present, there are two problems in the clinical practice of MTX in the field of rheumatic immunity. On the one hand, MTX is not fully used, especially clinicians who are not familiar with the off-label use of the drug, do not understand the therapeutic value of MTX, or know it but dare not use it because the drug label has no indication. On the other hand, the use of MTX is not standardized, such as insufficient dose, not titrated to large dose/optimal dose, treatment when oral intolerance occurs. Therefore, it is necessary to form an expert consensus on the off-label use of MTX in the treatment of rheumatoid immune diseases, in order to further standardize clinical application and strengthen pharmaceutical management.