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

Abstract

BackgroundNeuromyelitis 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.ObjectiveTo assess the long-term safety and efficacy in NMOSD patients receiving maintenance therapy with B-cell-depleting agent rituximab for more than 2years.MethodNMOSD 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.ResultsA total of 44 NMOSD patients were included, of those 30 were treatment naive (68). The mean age was 37.2years with 79.5 females. With overall observation period of 31.67.3months (24-48months), 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 p id=Par5 Rituximab treatment is well tolerated, safe, and efficacious with a minor risk of mild infusion reactions for NMOSD patients.