A recent interim analysis of a post-marketing surveillance study in Japan has shown promising results for the use of eculizumab in patients with aquaporin-4 antibody-positive (AQP4+) neuromyelitis optica spectrum disorder (NMOSD), according to a study recently presented as a poster in the 9th Congress of the European Academy of Neurology, and published in the European Journal of Neurology.

“The observed reduction in IST use, also aligned with other real-world experiences, underlines the benefits of C5 inhibition in these patients,” the authors wrote.

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The study, conducted from November 2019 to October 2022, involved 178 patients, with 89 patients included in the safety set. Out of the safety set, 62 adverse events (AEs) were reported, with 25 deemed treatment-related. No meningococcal infections occurred during the study.

In terms of effectiveness, the relapse rate decreased significantly during eculizumab treatment, with only 1 relapse observed during the 89.42 patient-years of treatment, compared to a relapse rate of 0.68/patient-year before starting eculizumab.

Before initiating eculizumab, a significant proportion of patients (53.5%) received immunosuppressant therapy (IST). This number decreased to 44.6% in the 6-12 months following eculizumab treatment. Additionally, the proportion of patients taking prednisolone at a dosage higher than 10 mg/day showed a notable decrease from 44.2% before eculizumab treatment to 18.2% and 11.2% at 52 to 56 and 100 to 104 weeks after treatment, respectively.

The findings of this real-world study align with those from the PREVENT study, further confirming the efficacy and tolerability of eculizumab in Japanese patients with AQP4+ NMOSD.

These positive results provide valuable insights into eculizumab’s long-term safety and effectiveness in a real-world setting, reinforcing its role as a crucial treatment option for AQP4+ NMOSD, the researchers noted.

As further research continues to explore the potential benefits of eculizumab, healthcare professionals can feel more confident in their ability to provide optimal care to patients with this disorder, the study team concluded.


Nakahara J, Nakashima I, Yokote H, et al. Eculizumab in AQP4+ neuromyelitis optica spectrum disorder: 3 years of data from Japanese post-marketing surveillance. Eur Neurol. Published online June 30, 2023.