The real-world experience of biologics for neuromyelitis optica spectrum disorders as first-line and switched therapy.

BACKGROUND

Several biologics have demonstrated the efficacy in reducing relapses of neuromyelitis optica spectrum disorder (NMOSD) with aquaporin-4 immunoglobulin G (AQP4-IgG). However, real-world data on biologics for NMOSD are limited. We aimed to evaluate the efficacy and safety of biologics in clinical practice.

METHODS

We retrospectively reviewed the medical records of 117 patients with AQP4-IgG-positive NMOSD in our hospitals between 2000 and 2024. Patients were classified into Biologics-1st and glucocorticoids/immunosuppressants (GC/IS)-1st by their first-line therapy, and then those who switched from GC/IS to biologics (Switched to Biologics). Annualized relapse rate (ARR) and changes in Expanded Disability Status Scale (EDSS) scores were analyzed, and the safety was assessed in all biologics-treated patients.

RESULTS

Among 45 patients who switched to biologics, significant reductions were observed in ARR [median (interquartile range, IQR), 0.0 (0.0-0.0) vs. 0.4 (0.1-0.6), p < 0.0001] and annual changes in EDSS scores [median (IQR), 0.0 (0.0-0.0) vs. 0.1 (0.0-0.3), p < 0.0001] after switching. No relapses occurred during 24.3 patient-years in Biologics-1st group, while GC/IS-1st group had 330 relapses during 970.3 patient-years (p = 0.0031). The ARR was significantly lower in Biologics-1st than in GC/IS-1st [median (IQR), 0.00 (0.00-0.00) vs. 0.20 (0.10-0.50), p < 0.0001], while the EDSS score change was comparable. The safety profile of biologics was generally consistent with the clinical trial data.

CONCLUSIONS

Biologics were highly efficacious in preventing relapses of NMOSD with AQP4-IgG in real-world clinical practice.