title: Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients.
abstract: 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.
  
  
  METHODS
  Retrospective case series with prospective telephone follow-up.
  
  
  METHODS
  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).
  
  
  METHODS
  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.
  
  
  CONCLUSIONS
  Mycophenolate is associated with reduction in relapse frequency and stable or reduced disability in patients with NMO spectrum disorders.
authors.affiliation: Department of Neurology, Mayo Clinic College of Medicine, Rochester, Minnesota 55905, USA.
authors.forename: Anu
authors.initials: A
authors.lastname: Jacob
authors.forename: Marcelo
authors.initials: M
authors.lastname: Matiello
authors.forename: Brian G
authors.initials: BG
authors.lastname: Weinshenker
authors.forename: Dean M
authors.initials: DM
authors.lastname: Wingerchuk
authors.forename: Claudia
authors.initials: C
authors.lastname: Lucchinetti
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Shuster
authors.forename: Jonathan
authors.initials: J
authors.lastname: Carter
authors.forename: B Mark
authors.initials: BM
authors.lastname: Keegan
authors.forename: Orhun H
authors.initials: OH
authors.lastname: Kantarci
authors.forename: Sean J
authors.initials: SJ
authors.lastname: Pittock
mesh_terms: D000328:Adult; D000368:Aged; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000008:administration & dosage* / Q000009:adverse effects; D051401:Aquaporin 4 / Q000276:immunology / Q000378:metabolism; D004185:Disability Evaluation; D064420:Drug-Related Side Effects and Adverse Reactions; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007074:Immunoglobulin G / Q000276:immunology; D007165:Immunosuppression Therapy / Q000379:methods*; D007407:Interviews as Topic; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*; D009471:Neuromyelitis Optica / Q000188:drug therapy* / Q000276:immunology* / Q000503:physiopathology; D011446:Prospective Studies; D012189:Retrospective Studies; D055502:Secondary Prevention; D016896:Treatment Outcome
chemical_list: C496030:AQP4 protein, human; D000894:Anti-Inflammatory Agents, Non-Steroidal; D051401:Aquaporin 4; D007074:Immunoglobulin G; D009173:Mycophenolic Acid