Monoclonal antibodies in neuromyelitis optica spectrum disorder: a systematic review and meta-analysis

Neuromyelitis optica spectrum disorder (NMOSD) is a severe central nervous system (CNS) inflammatory disease. New immunobiological therapies have emerged as promising options. Therefore, this study aimed to conduct a systematic review and meta-analysis of monoclonal antibodies for NMOSD treatment. This review was registered in PROSPERO (ID CRD420251106764) and was conducted in accordance with standard guidelines. We included randomized clinical trials that analyzed immunobiological therapies for NMOSD. Data were extracted using EndNote and Excel. Risk of bias was assessed with the Cochrane RoB 2.0 tool. A random-effects meta-analysis was performed for the main outcomes. Publication bias was evaluated. Eight studies met the eligibility criteria and were included in the final analysis. All were rated as presenting some concerns regarding risk of bias. Our analysis demonstrated a significant reduction in relapse risk (HR 0.21; 95% CI 0.12 to 0.39, NNT = 3), reduction in EDSS (SMD − 0.33; 95% CI -0.54 to -0.13), with no differences in severe adverse events (RR 0.89; 95% CI 0.57 to 1.41, NNH = 100) between the monoclonal antibody group and controls. Our meta-analysis demonstrated that monoclonal antibodies are effective and safe, significantly reducing relapse risk and disability without increasing severe adverse events.