High‐Efficacy Treatment in Neuromyelitis Optica Specturm Disorder Patients With Seropositive AQP4 Antibodies—A Real‐World Study

ABSTRACT Objective To compare the effectiveness of high‐efficacy treatments (HET) and low‐efficacy treatments (LET) in NMOSD patients with anti‐aquaporin‐4 antibodies (AQP4‐ab). Methods In this multi‐center study, we analyzed 183 AQP4‐ab seropositive NMOSD patients who received immunosuppressive treatments (IST). Primary outcomes included annualized relapse rate (ARR) and Expanded Disability Status Scale (EDSS). Results A total of 86 HET and 143 LET treatment episodes were included. Patients with HET had the lower relapse rate (41.9% vs. 59.4%, p = 0.015). At the final follow‐up, the HET group had lower ARR ( p = 0.003) and EDSS ( p = 0.004) compared to LET. HET ( p < 0.001, adjusted p < 0.001), early IST initiation after onset ( p = 0.007, adjusted p = 0.008) and younger age of onset ( p = 0.024, adjusted p = 0.028) were the protective factors for high EDSS, and HET (HR: 0.66, 95% CI: 0.44–0.99, p = 0.047) prolonged the remission after IST. Meantime, Anderson‐Gill analysis indicated that HET is associated with a lower risk of relapse ( p < 0.001). After PSM, patients receiving HET had significantly reduced ARR ( p = 0.046) and EDSS scores ( p =0.030) compared to those receiving LET. Interpretation Our findings indicated the superior efficacy in reducing neurological disability and relapse risk of HET in AQP4‐ab seropositive NMOSD patients.