Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients

Arch Neurol. 2009 Sep;66(9):1128-33. doi: 10.1001/archneurol.2009.175.


Background: Neuromyelitis optica (NMO) is the first inflammatory autoimmune demyelinating disease of the central nervous system for which a specific antigenic target has been identified; the marker autoantibody NMO-IgG specifically recognizes the astrocytic water channel aquaporin 4. Current evidence strongly suggests that NMO-IgG may be pathogenic. Since disability accrues incrementally related to attacks, attack prevention with immunosuppressive therapy is the mainstay of preventing disability.

Objective: To evaluate the efficacy and safety of mycophenolate mofetil therapy in NMO spectrum disorders.

Design: Retrospective case series with prospective telephone follow-up.

Setting: Mayo Clinic Health System. Patients Twenty-four patients with NMO spectrum disorders (7 treatment-naive). Intervention Mycophenolate mofetil (median dose of 2000 mg per day).

Main outcome measures: Annualized relapse rates and disability (Expanded Disability Status Scale).

Results: At a median follow-up of 28 months (range, 18-89 months), 19 patients (79%) were continuing treatment. The median duration of treatment was 27 months (range, 1-89 months). The median annualized posttreatment relapse rate was lower than the pretreatment rate (0.09; range, 0-1.5; and 1.3; range, 0.23-11.8, respectively; P < .001). Disability stabilized or decreased in 22 of 24 patients (91%). One patient died of disease complications during follow-up. Six patients (25%) noted adverse effects during treatment with mycophenolate.

Conclusion: Mycophenolate is associated with reduction in relapse frequency and stable or reduced disability in patients with NMO spectrum disorders.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Anti-Inflammatory Agents, Non-Steroidal / administration & dosage*
  • Anti-Inflammatory Agents, Non-Steroidal / adverse effects
  • Aquaporin 4 / immunology
  • Aquaporin 4 / metabolism
  • Disability Evaluation
  • Drug-Related Side Effects and Adverse Reactions
  • Female
  • Follow-Up Studies
  • Humans
  • Immunoglobulin G / immunology
  • Immunosuppression Therapy / methods*
  • Interviews as Topic
  • Male
  • Middle Aged
  • Mycophenolic Acid / administration & dosage
  • Mycophenolic Acid / adverse effects
  • Mycophenolic Acid / analogs & derivatives*
  • Neuromyelitis Optica / drug therapy*
  • Neuromyelitis Optica / immunology*
  • Neuromyelitis Optica / physiopathology
  • Prospective Studies
  • Retrospective Studies
  • Secondary Prevention
  • Treatment Outcome


  • AQP4 protein, human
  • Anti-Inflammatory Agents, Non-Steroidal
  • Aquaporin 4
  • Immunoglobulin G
  • Mycophenolic Acid