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

Arch Neurol. 2008 Nov;65(11):1443-8. doi: 10.1001/archneur.65.11.noc80069. Epub 2008 Sep 8.


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.

Publication types

  • Multicenter Study
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antibodies, Monoclonal / administration & dosage
  • Antibodies, Monoclonal / adverse effects
  • Antibodies, Monoclonal / therapeutic use*
  • Antibodies, Monoclonal, Murine-Derived
  • Child
  • Disability Evaluation
  • Drug Administration Schedule
  • Female
  • Humans
  • Immunologic Factors / administration & dosage
  • Immunologic Factors / adverse effects
  • Immunologic Factors / therapeutic use*
  • Longitudinal Studies
  • Male
  • Middle Aged
  • Neuromyelitis Optica / drug therapy*
  • Neuromyelitis Optica / mortality
  • Neuromyelitis Optica / physiopathology
  • Retrospective Studies
  • Rituximab
  • Secondary Prevention
  • Treatment Outcome
  • Young Adult


  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Murine-Derived
  • Immunologic Factors
  • Rituximab