Long-term tolerability, safety and efficacy of rituximab in neuromyelitis optica spectrum disorder: a prospective study



Neuromyelitis optica spectrum disorder (NMOSD) is a B-cell-mediated disease with autoimmunity towards the astrocyte water channel aquaporin-4 (AQP-4) in the central nervous system.


To assess the long-term safety and efficacy in NMOSD patients receiving maintenance therapy with B-cell-depleting agent rituximab for more than 2 years.


NMOSD patients were included prospectively from 2014 to 2018 and received continuous cycles of rituximab infusions biannually. Incidence of adverse events (AE), serious AEs (SAE), and infusion-related AEs were evaluated through monthly phone calls and neurological examination every 4 months.


A total of 44 NMOSD patients were included, of those 30 were treatment naive (68%). The mean age was 37.2 years with 79.5% females. With overall observation period of 31.6 ± 7.3 months (24–48 months), tolerability was assessed as satisfactory in most cases. We observed infusion reactions (mostly mild) in 31.8% of patients and 31.8% never experienced any AEs after a mean 5.1 cycles of rituximab therapy. Rituximab was also beneficial in terms of improvement in relapse rate (from 0.26 ± 0.54 to 0, P = 0.003) and Expanded Disability Status Scale (from 4.1 ± 1.8 to 3.1 ± 1.8, P < 0.001). Stratification according to AQP4-IgG serostatus showed no difference between groups.


Rituximab treatment is well tolerated, safe, and efficacious with a minor risk of mild infusion reactions for NMOSD patients.

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The authors wish to thank the patients for their cooperation throughout the follow-up process.


No funding received.

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Corresponding author

Correspondence to N. Asgari.

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The authors declare that they have no conflict of interest.

Ethical standard

The study was approved by research ethics committee of Isfahan University of Medical Sciences (Approval Code: IR.MUI.RSEARCH.REC.1397.171) and it was performed in accordance with the ethical standards laid down in the 1964 Declaration of Helsinki and its later amendments.

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Written informed consents were obtained from all patients upon enrollment.

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Shaygannejad, V., Fayyazi, E., Badihian, S. et al. Long-term tolerability, safety and efficacy of rituximab in neuromyelitis optica spectrum disorder: a prospective study. J Neurol 266, 642–650 (2019). https://doi.org/10.1007/s00415-019-09180-9

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  • Neuromyelitis optica spectrum disease
  • Rituximab
  • Tolerability
  • Safety
  • Efficacy
  • Adverse drug reactions