Real-World Efficacy and Safety of Neuromyelitis Optica Spectrum Disorder Disease-Modifying Treatments
Journal: Neurology Neuroimmunology & Neuroinflammation; January 6, 2026
Author(s): Philippe A Bilodeau, Mattia Wruble Clark, Avanteeka Ganguly, Jenna Bernice Harowitz, Joao Vitor Mahler, Mulan Jiang, Sathya S Narasimhan, Danielle Kei Pua, Brian C Healy, Farrah Jasmine Mateen, Michael Levy, Shamik Bhattacharyya
How do different treatments for NMOSD compare in real-world settings?
This study looked at how well different treatments, including the more traditionally used treatments such as rituximab, mycophenolate mofetil (MMF), and azathioprine, and newer approved drugs—eculizumab, inebilizumab, and satralizumab—work for NMOSD treatment in real-world clinical practice, and how safe they are.
The researchers studied 176 patients with NMOSD who were treated at one hospital network and checked on for about 9 years, which involved assessing data from a total of 691 relapses. The average age at the time of the first attack was 42 years, and 85% of the patients were female.
The study found that the newer, approved NMOSD treatments, namely, eculizumab, inebilizumab, and satralizumab, were generally more effective at preventing relapses than rituximab, azathioprine, and MMF. Among all treatments, a newer class of drugs called C5 inhibitors (example, eculizumab and ravulizumab) resulted in the lowest relapse rates, while azathioprine resulted in the highest relapse rates. C5 inhibitors also had the lowest risk of serious infections.
In terms of overall results as a combination of preventing relapses and side effects, azathioprine and MMF performed worse than rituximab.
Overall, the study suggests that newer, approved treatments for NMOSD may be more effective and safer than some older or commonly used treatments in real-world settings.
Related article: Real-world multicentre cohort study on choices and effectiveness of immunotherapies in NMOSD and MOGAD
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