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

Abstract

Background

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.

Objective

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.

Method

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.

Results

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.

Conclusion

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

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Acknowledgements

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

Funding

No funding received.

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Corresponding author

Correspondence to N. Asgari.

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Conflicts of interest

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.

Informed consent

Written informed consents were obtained from all patients upon enrollment.

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Cite this article

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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Keywords

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