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

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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.

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