Abstract
BACKGROUND: This study aimed to compare the real-world efficacy and safety of rituximab and inebilizumab in patients with aquaporin-4 immunoglobulin G (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder (NMOSD). METHODS: This retrospective study included patients treated with rituximab or inebilizumab at two tertiary hospitals in China between January 2015 and June 2025. Propensity score matching was conducted to reduce between-group imbalance. The primary endpoint was time to first confirmed relapse. Secondary outcomes included changes in annualized relapse frequency, EDSS scores, and serum IgG and AQP4-IgG levels. Safety profiles were also assessed. RESULTS: A total of 276 patients were analyzed (rituximab: 211; inebilizumab: 65), yielding 61 well-balanced pairs after propensity score matching. In terms of efficacy, clinical outcomes and AQP4-IgG dynamics were comparable between groups in pre- and post-matching analyses, although inebilizumab exhibited a significantly greater reduction in serum IgG levels at 6 months. Safety profiles differed. Rituximab was associated with a higher incidence of overall adverse events, driven primarily by infusion-related reactions, whereas other adverse events remained comparable. CONCLUSIONS: In our real-world study of 276 patients, rituximab and inebilizumab demonstrated comparable efficacy in the medium term but differed in their safety profiles, with a significantly higher incidence of infusion-related reactions observed in the rituximab group.