Efficacy and safety of azathioprine for neuromyelitis optica spectrum disorders: a meta-analysis of real-world studies

Abstract Objective This study aimed to perform a meta-analysis of the efficacy and safety of azathioprine (AZA) for neuromyelitis optica spectrum disorders (NMOSD), considering the potential predictive factors related to patient response to AZA in this disease. Methods We performed a systematic online query in PubMed, EMBASE, The Cochrane Library, ClinicalTrials.gov, China National Knowledge Infrastructure, WANFANG DATA, and CQVIP DATA. The available studies on the use of AZA in NMOSD patients were included. Results We analyzed a total of 21 studies including 1016 patients. Results demonstrated that AZA significantly decreased annual relapse rate (ARR) by 1.164 (95% confidence intervals (CI), -1.396 to -0.932; p Conclusion AZA is effective in reducing relapse and improving patients’ neurological function. However, liver function monitoring and routine blood monitoring remain necessary. Within the safe upper limit, a higher dose of AZA may be associated with a better efficacy for NMOSD.