DOI: 10.1177/17562864261500513 ISSN: 1756-2864

Real-world effectiveness and safety of satralizumab in AQP4-IgG-seropositive neuromyelitis optica spectrum disorder: Across different clinical scenarios

Yang Ji, JuanJuan Zhang, Jing Du, Ling Wei, Jun Zhang, Chenyi Shi, Tieyu Wu, Yan Chen, Xiaokun Qi, Qun Xue, Yanghua Tian, Shugang Cao
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Background

Satralizumab, an interleukin-6 receptor monoclonal antibody, is approved for aquaporin-4 IgG (AQP4-IgG)-seropositive neuromyelitis optica spectrum disorder (NMOSD). However, real-world evidence remains limited, particularly regarding treatment initiation during acute attacks, transitions from B-cell-depleting therapies, and safety during pregnancy.

Objectives

To evaluate the real-world effectiveness and safety of satralizumab in AQP4-IgG–seropositive NMOSD and to assess outcomes across key clinical scenarios.

Design

A multicenter retrospective observational study.

Methods

This study evaluated 36 patients with AQP4-IgG-seropositive NMOSD treated with satralizumab. Primary clinical evaluations included changes in the annualized relapse rate (ARR), and Expanded Disability Status Scale (EDSS) scores, alongside safety profiles. Special clinical scenarios, including the feasibility of initiation during acute attacks, outcomes in patients with persistent disease activity despite prior B-cell-depleting therapies, and exposure during pregnancy, were also evaluated.

Results

Over a median follow-up of 16.7 months, 97.2% of patients remained relapse-free. The overall median ARR decreased from 0.3 to 0.0 ( p < 0.001), and the median EDSS score decreased by 0.5 points ( p = 0.001). Patients initiating satralizumab during acute attacks (n = 10) demonstrated significantly greater EDSS score reductions compared with those initiating during remission (n = 26) (median change: -1.5 vs 0.0, p < 0.001), with no significant difference in ARR reductions. Furthermore, 6 patients with persistent disease activity on prior B-cell-depleting therapies achieved sustained clinical stability after transitioning to satralizumab. Adverse events occurred in 22.2% of patients, predominantly comprising mild upper respiratory tract infections. Notably, 1 patient exposed to satralizumab during early pregnancy remained relapse-free without severe maternal or obstetric complications; the neonate developed mild pneumonia and a 2-mm atrial septal defect, both of which were managed conservatively.

Conclusion

Satralizumab maintained low relapse rates and facilitated EDSS score reductions, even following B-cell-depleting therapy failure. Pronounced EDSS improvements with acute-phase initiation likely reflect concomitant rescue therapies and natural recovery. Single-case pregnancy exposure provides preliminary safety insights, requiring broader registry validation. These findings expand real-world evidence for satralizumab across diverse clinical scenarios.

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