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. 2025 Mar;21(2):131-136.
doi: 10.3988/jcn.2024.0485.

Evaluating Rituximab Failure Rates in Neuromyelitis Optica Spectrum Disorder: A Nationwide Real-World Study From South Korea

Affiliations

Evaluating Rituximab Failure Rates in Neuromyelitis Optica Spectrum Disorder: A Nationwide Real-World Study From South Korea

Su-Hyun Kim et al. J Clin Neurol. 2025 Mar.

Abstract

Background and purpose: Treatments for neuromyelitis optica spectrum disorder (NMOSD) such as eculizumab, ravulizumab, satralizumab, and inebilizumab have significantly advanced relapse prevention, but they remain expensive. Rituximab is an off-label yet popular alternative that offers a cost-effective solution, but its real-world efficacy needs better quantification for guiding the application of newer approved NMOSD treatments (ANTs). This study aimed to determine real-world rituximab failure rates to anticipate the demand for ANTs and aid in resource allocation.

Methods: We conducted a nationwide retrospective study involving 605 aquaporin-4-antibody-positive NMOSD patients from 22 centers in South Korea that assessed the efficacy and safety of rituximab over a median follow-up of 47 months.

Results: The 605 patients treated with rituximab included 525 (87%) who received continuous therapy throughout the follow-up period (median=47 months, interquartile range=15-87 months). During this period, 117 patients (19%) experienced at least 1 relapse. Notably, 68 of these patients (11% of the total cohort) experienced multiple relapses or at least 1 severe relapse. Additionally, 2% of the patients discontinued rituximab due to adverse events, which included severe infusion reactions, neutropenia, and infections.

Conclusions: This study has confirmed the efficacy of rituximab in treating NMOSD, as evidenced by an 87% continuation rate among patients over a 4-year follow-up period. Nevertheless, the occurrence of at least one relapse in 19% of the cohort, including 11% who experienced multiple or severe relapses, and a 2% discontinuation rate due to adverse events highlight the urgent need for alternative therapeutic options.

Keywords: eculizumab; inebilizumab; neuromyelitis optica; rituximab; satralizumab.

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Conflict of interest statement

S-HK has lectured, consulted, and received honoraria from Bayer Schering Pharma, Biogen, Genzyme, Merck Serono, and UCB and received a grant from the National Cancer Center of Korea. JHM is funded by and has received research support from the National Research Foundation of Korea (MIST and KHIDI) and SMC Research and Development Grant. She has lectured, consulted, and received honoraria from Bayer Healthcare, Merk, Biogen Idec, Sanofi, UCB, Samsung Bioepis, Mitsubishi Tanabe, Celltrion, Roche, Janssen, AstraZeneca, and MedicinePharma. S-HK, J-HM, SMK, E-JL, Y-ML, YNK, ES, SK (Sooyoung Kim), MSP, T-SN, B-AY, JKK, KJS, YHK, JMS, JBB, SK (Sohyeon Kim), HYS, S-YO, OK, SK (Sunyoung Kim), SYL, N-HK, EBC, S-YK, SO, JSB, S-WA, KHK, YRK, WJ, SHC, YHC, and JWH report no financial disclosures. HYS received a grant from the National Research Foundation of Korea and research support from Hanmi, Myung In Pharm, and Yuhan; received consultancy/speaker fees from Alexion, Astellas, AstraZeneca, Biogen, Daewoong Pharmaceutical, Eisai, Euroimmun, GC Pharma, Genuv, Genzyme, Handok Pharmaceutical, Janssen, Merck Serono, Mitsubishi Tanabe Pharma, Roche, Sanofi, and UCB; is an associated editor for the Journal of Clinical Neurology. HJK received a grant from the National Research Foundation of Korea and research support from Aprilbio and Eisai; received consultancy/speaker fees from Alexion, Aprilbio, Altos Biologics, Biogen, Celltrion, Daewoong, Eisai, GC Pharma, Handok, Horizon Therapeutics, Kaigene, Kolon Life Science, MDimune, Mitsubishi Tanabe Pharma, Merck Serono, Novartis, Roche, Sanofi Genzyme, Teva-Handok, and UCB; is a co-editor for the Multiple Sclerosis Journal and an associated editor for the Journal of Clinical Neurology. E-JL and KJS are editorial board members of Journal of Clinical Neurology. However, they were not involved in the peer review or decision-making process for this manuscript. All other authors report no potential conflicts of interest related to this study.

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