Treatment of neuromyelitis optica with rituximab: retrospective analysis of 25 patients

Abstract

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.