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The following is a summary of “Eculizumab Use in Neuromyelitis Optica Spectrum Disorders: Routine Clinical Care Data from a European Cohort,” published in the October 2024 issue of Neurology by Ringelstein et al.
Neuromyelitis optica spectrum disorders (NMOSDs) are managed by preventing attacks, with eculizumab (ECU), a terminal complement cascade inhibitor, shown to be effective in aquaporin-4 (AQP4)-IgG seropositive (+) cases in clinical trials.
Researchers conducted a retrospective study evaluating the effectiveness and safety of eculizumab in patients with AQP4-IgG+ NMOSD.
They reviewed data of patients with AQP4-IgG+ NMOSD treated with ECU (December 2014 and April 2022) at 21 centers in Germany and Austria. Primary outcomes included annualized attack rate (AAR), MRI activity, Expanded Disability Status Scale (EDSS), and safety.
The results showed 52 patients (87% female, age 55.0 ± 16.3 years) received ECU for a median of 16.2 months (IQR 9.6 – 21.7). After meningococcal vaccination, 7 of 36 patients (19%) had attacks, while no attacks occurred in those on prednisone. Eculizumab reduced the median AAR from 1.0 to 0 (P<0.001), and MRI activity and EDSS remained stable. Serious infections occurred in 13% of patients, and 10% died (primarily older, severely disabled).
They concluded that ECU effectively prevented NMOSD attacks, though risks such as infections and post-vaccination attacks must be considered, especially in patients with comorbidities.