Efficacy and Tolerability of Azathioprine for Neuromyelitis Optica Spectrum Disorder: A Systematic Review and Meta-Analysis

The systematic review and meta-analysis concluded that azathioprine (AZA) was less effective than rituximab for treating neuromyelitis optica spectrum disorder (NMOSD), with limited efficacy data compared to other drugs like mycophenolate mofetil (MMF), cyclophosphamide, and interferon-β. The study included 9 relevant studies with a total of 977 patients. AZA was associated with frequent adverse events, including elevated liver enzymes, leukopenia, and hair loss, which could lead to medication noncompliance. Despite improving relapses and disability, AZA's adverse effects suggest it should be considered a second-line treatment. More well-conducted clinical trials were recommended to better establish AZA's benefits and risks.