Treatment of Neuromyelitis Optica With Rituximab Retrospective Analysis of 25 Patients

Background: Neuromyelitis optica (NMO) is an uncommon, life-threatening inflammatory demyelinating disorder. Recently, much has become known about its immunopathogenesis. However, optimal treatments, with expected outcomes, have not been established. Objective: To evaluate the use and efficacy of rituximab for treating NMO. Design: Retrospective multicenter case series of NMO patients treated with rituximab. Setting: Seven tertiary medical centers in the United States and England. Patients: Twenty-five patients (including 2 children), 23 of whom experienced relapses despite use of other drugs before rituximab. Extended follow-up of 7 previously reported patients is included. Interventions: Infusions of rituximab at median intervals of 8 months. Main Outcome Measures: Annualized relapse rate and disability (expressed as Expanded Disability Status Scale score). Results: At a median follow-up of 19 months, the median annualized posttreatment relapse rate was lower than the pretreatment rate (0 [range 0-3.2] vs 1.7 [range, 0.5-5] relapses, P<.001). Disability improved or stabilized in 20 of 25 patients (80%, P=.02). Two patients died during the follow-up period, 1 owing to a brainstem relapse and 1 owing to suspected septicemia. Infections were reported in 20% of patients. Conclusions: In NMO, treatment with rituximab appears to reduce the frequency of attacks, with subsequent stabilization or improvement in disability.