2023 American Academy of Neurology Abstract Website

Ilya Ayzenberg¹, Susanna Asseyer², Gero Lindenblatt³, Katinka Fischer⁴, Refik Pul⁶, Sinem-Hilal Özalp⁶, Lisa Lohmann⁷, Katrin Giglhuber⁸, Vivien Häussler⁹, Michael Karenfort⁵, Kerstin Hellwig¹, Friedemann Paul², Judith Bellmann-Strobl², Carolin Otto², Klemens Ruprecht², Tjalf Ziemssen¹⁰, Alexander Emmer¹¹, Veit Rothhammer¹², Florian Nickel¹³, Klemens Angstwurm¹⁴, Ralf Linker¹⁴, Clemens Warnke¹⁵, Sven Jarius¹⁶, Mirjam Korporal-Kuhnke¹⁶, Brigitte Wildemann¹⁶, Stephanie Wolff¹⁷, Maria Seipelt¹⁸, Yavor Yalachkov¹⁹, Nele Retzlaff²⁰, Uwe Zettl²⁰, Paulus Rommer²¹, Markus Kowarik²², Jonathan Wickel²³, Christian Geis²³, Martin W. Hümmert²⁴, Corinna Trebst²⁴, Makbule Senel²⁵, Luisa Klotz⁷, Ralf Gold¹, Christoph Kleinschnitz⁶, Sven Meuth⁴, Orhan Aktas⁴, Achim Berthele⁸, Marius Ringelstein⁴
¹St.Josef Hospital, Department of Neurology, Ruhr-Universität Bochum, ²Charite Universitätsmedizin in Berlin, ³Department of Neurology, Johanna Etienne Hospital, ⁴Department of Neurology, ⁵Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Children's Hospital, Medical Faculty, Heinrich-Heine-University Düsseldorf, ⁶Department of Neurology, University Medicine Essen, University of Duisburg-Essen, ⁷Department of Neurology, University Hospital Münster, ⁸Department of Neurology, School of Medicine, Technical University Munich, Klinikum rechts der Isar, ⁹Department of Neurology and Institute of Neuroimmunology and MS (INIMS), University Medical Center Hamburg-Eppendorf, ¹⁰University Clinic Dresden, ¹¹Department of Neurology, University Hospital Halle, ¹²University Hospital Erlangen, ¹³Department of Neurology, University Hospital Erlangen, ¹⁴Department of Neurology, University of Regensburg, ¹⁵Department of Neurology, University Hospital Köln, ¹⁶Molecular Neuroimmunology Group, Department of Neurology, University of Heidelberg, ¹⁷Department of Neurology, University of Giessen, ¹⁸Department of Neurology, University Hospital Marburg, ¹⁹Department of Neurology, University Hospital Frankfurt, ²⁰Department of Neurology, University Hospital Rostock, ²¹Department of Neurology, Medical University of Vienna, ²²Department of Neurology, University Hospital Tübingen, ²³Section of Translational Neuroimmunology, Department of Neurology, Jena University Hospital, ²⁴Department of Neurology, Hannover Medical School, ²⁵Department of Neurology, University Hospital Ulm

Objective:

To evaluate efficacy and safety of eculizumab (ECU) in a large real-world Neuromyelitis optica spectrum disorder (NMOSD) cohort.

Background:

Real-world data on Eculizumab in NMOSD are still scarce. The drug was highly effective in preventing relapses in the phase-III PREVENT trial and is approved for AQP4-IgG+ NMOSD, but carries potential risk of life-threatening meningococcal infection. Therefore, appropriate vaccination or antibiotic prophylaxis are mandatory.

Design/Methods:

In this retrospective multicenter study we analyzed annualized relapse rate (ARR), disability (EDSS) and rate of serious infections during ECU, as well as tolerability of meningococcal vaccination.

Results:

55 patients (52 AQP4-IgG+, 84% female, 48.6±16.6 years) with an active NMOSD (median ARR 1.0, EDSS 6.0) were included. 13/55 were treatment-naive and 42/55 switched from another immunotherapy. 38/55 experienced a relapse or asymptomatic MRI activity <3 months before ECU. During ECU-treatment (17.4±15.4 months) ARR dropped significantly (from 1.0(0-3.0) to 0(0-1.4), p<0.001), 86% of patients were relapse-free. The median EDSS remained stable.

39/55 patients received at least one meningococcal vaccination prior-to-ECU, resulting in a significant treatment delay comparing to those with antibiotic prophylaxis. 9/39 patients have been vaccinated despite concomitant oral prednisone and reported no relapses. In contrast 10/30 (33%) patients vaccinated prior-to-ECU without prednisone experienced new attacks shortly thereafter (interval 9.5±7.7 days). None of 24/55 vaccinated during ECU relapsed during following 4 weeks.

Despite vaccination one patient developed meningococcal sepsis, 4 other multimorbid patients experienced non-meningococcal systemic infections. Five patients died during ECU, three in the context of sepsis and two due to myocardial infarction and ileus, respectively. Ten further patients discontinuated ECU due to different reasons.

Conclusions:

Real-world data confirm high efficacy of eculizumab in NMOSD. Risk of relapses in patients vaccinated prior to eculizumab start and potential severe infections in multimorbid patients must be considered. Further studies are needed to establish an optimal vaccination schedule in active NMOSD.