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Retrospective Analysis of 24 Patients

Anu Jacob, MD; Marcelo Matiello, MD; Brian G. Weinshenker, MD; Dean M. Wingerchuk, MD; Claudia Lucchinetti, MD; Elizabeth Shuster, MD; Jonathan Carter, MD; B. Mark Keegan, MD; Orhun H. Kantarci, MD; Sean J. Pittock, MD

Arch Neurol. 2009;66(9):1128-1133.

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.

Author Affiliations: Departments of Neurology, Mayo Clinic College of Medicine, Rochester, Minnesota (Drs Jacob, Matiello, Weinshenker, Lucchinetti, Keegan, Kantarci, and Pittock); Mayo Clinic, Scottsdale, Arizona (Drs Wingerchuk and Carter); and Mayo Clinic, Jacksonville, Florida (Dr Shuster). Dr Jacob is currently a consultant at the Walton Centre, Liverpool, England.

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