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| Item Type: | Article |
|---|---|
| Title: | Eculizumab use in neuromyelitis optica spectrum disorders: routine clinical care data from a european cohort |
| Creators Name: | Ringelstein, M., Asseyer, S., Lindenblatt, G., Fischer, K., Pul, R., Skuljec, J., Revie, L., Giglhuber, K., Häußler, V., Karenfort, M., Hellwig, K., Paul, F., Bellmann-Strobl, J., Otto, C., Ruprecht, K., Ziemssen, T., Emmer, A., Rothhammer, V., Nickel, F.T., Angstwurm, K., Linker, R., Laurent, S.A., Warnke, C., Jarius, S., Korporal-Kuhnke, M., Wildemann, B., Wolff, S., Seipelt, M., Yalachkov, Y., Retzlaff, N., Zettl, U.K., Rommer, P.S., Kowarik, M.C., Wickel, J., Geis, C., Hümmert, M.W., Trebst, C., Senel, M., Gold, R., Klotz, L., Kleinschnitz, C., Meuth, S.G., Aktas, O., Berthele, A. and Ayzenberg, I. |
| Abstract: | BACKGROUND AND OBJECTIVES: Attack prevention is crucial in managing neuromyelitis optica spectrum disorders (NMOSDs). Eculizumab (ECU), an inhibitor of the terminal complement cascade, was highly effective in preventing attacks in a phase III trial of aquaporin-4 (AQP4)-IgG seropositive(+) NMOSDs. In this article, we evaluated effectiveness and safety of ECU in routine clinical care. METHODS: We retrospectively evaluated patients with AQP4-IgG+ NMOSD treated with ECU between December 2014 and April 2022 at 20 German and 1 Austrian university center(s) of the Neuromyelitis Optica Study Group (NEMOS) by chart review. Primary outcomes were effectiveness (assessed using annualized attack rate [AAR], MRI activity, and disability changes [Expanded Disability Status Scale {EDSS}]) and safety (including adverse events, mortality, and attacks after meningococcal vaccinations), analyzed by descriptive statistics. RESULTS: Fifty-two patients (87% female, age 55.0 ± 16.3 years) received ECU for 16.2 (interquartile range [IQR] 9.6 - 21.7) months. Forty-five patients (87%) received meningococcal vaccination before starting ECU, 9 with concomitant oral prednisone and 36 without. Seven of the latter (19%) experienced attacks shortly after vaccination (median: 9 days, IQR 6-10 days). No postvaccinal attack occurred in the 9 patients vaccinated while on oral prednisone before starting ECU and in 25 (re-)vaccinated while on ECU. During ECU therapy, 88% of patients were attack-free. The median AAR decreased from 1.0 (range 0-4) in the 2 years preceding ECU to 0 (range 0-0.8; p < 0.001). The EDSS score from start to the last follow-up was stable (median 6.0), and the proportion of patients with new T2-enhancing or gadolinium-enhancing MRI lesions in the brain and spinal cord decreased. Seven patients (13%) experienced serious infections. Five patients (10%; median age 53.7 years) died on ECU treatment (1 from myocardial infarction, 1 from ileus with secondary sepsis, and 3 from systemic infection, including 1 meningococcal sepsis), 4 were older than 60 years and severely disabled at ECU treatment start (EDSS score = 7). The overall discontinuation rate was 19%. DISCUSSION: Eculizumab proved to be effective in preventing NMOSD attacks. An increased risk of attacks after meningococcal vaccination before ECU start and potentially fatal systemic infections during ECU-particularly in patients with comorbidities-must be considered. Further research is necessary to explore optimal timing for meningococcal vaccinations. CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that eculizumab reduces annualized attack rates and new MRI lesions in AQP4-IgG+ patients with NMOSD. |
| Keywords: | Humanized Monoclonal Antibodies, Aquaporin 4, Cohort Studies, Complement Inactivating Agents, Magnetic Resonance Imaging, Meningococcal Vaccines, Neuromyelitis Optica, Retrospective Studies, Treatment Outcome |
| Source: | Neurology |
| ISSN: | 0028-3878 |
| Publisher: | American Academy of Neurology |
| Volume: | 103 |
| Number: | 9 |
| Page Range: | e209888 |
| Date: | 12 November 2024 |
| Official Publication: | https://doi.org/10.1212/wnl.0000000000209888 |
| PubMed: | View item in PubMed |
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