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

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.