Affiliation:
1. Department of Neurology, Princess Alexandra Hospital, Woolloongabba, Australia
2. Department of Neurology, Royal Brisbane and Women's Hospital, Herston, Australia
3. University of Queensland Centre for Clinical Research, Royal Brisbane and Women's Hospital, Herston, Australia
Abstract
Background Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing, autoimmune, inflammatory astrocytopathy. Rituximab for B-cell suppression is a common treatment for NMOSD; however, large-scale randomised controlled trials are lacking. Objective Evaluate long-term efficacy and safety of rituximab for NMOSD. Methods Retrospective observational study of patients with NMOSD treated with rituximab. Annualised relapse rates (ARRs) before and during rituximab treatment were evaluated; Modified Rankin Scores (mRS) were measured as a marker of disability. Results In total, 37 patients were included: 27 aquaporin-4-IgG-seropositive and 10 seronegative NMOSD. The predominant rituximab dosing regimen was an initial 1000 mg, split over two 500 mg infusions, two weeks apart, followed by single 500 mg doses. Over a median follow-up of 54 months, ARR for the whole cohort was 0.136 (95% CI 0.088–0.201), significantly lower than the pretreatment ARR of 0.366 (95% CI 0.271–0.483, p < 0.001). There was a significant reduction in ARR for the seropositive subgroup, but not seronegative. Significant improvement in mRS was seen post-treatment. Infections were reported in 32% of patients during follow-up; most were mild. Conclusion Rituximab, at doses lower than traditionally used, may be an efficacious therapy for NMOSD, with a favourable safety profile.