title: Effectiveness of mycophenolate mofetil as first-line therapy in AQP4-IgG, MOG-IgG, and seronegative neuromyelitis optica spectrum disorders.
abstract: OBJECTIVE
  To evaluate the effectiveness and tolerance of mycophenolate mofetil (MMF) as a first-line treatment in neuromyelitis optica spectrum disorder (NMOSD).
  
  
  METHODS
  In all, 67 NMOSD patients treated by MMF as first-line therapy, from the NOMADMUS cohort were included. A total of 65 fulfilled 2015 NMOSD criteria, and 5 were myelin oligodendrocyte glycoprotein (MOG)-immunoglobulin G (IgG) positive. Effectiveness was evaluated on percentage of patients continuing MMF, percentage of patients free of relapse, pre- and post-treatment change in the annualized relapse rate (ARR), and Expanded Disability Status Scale (EDSS).
  
  
  RESULTS
  Among 67 patients, 40 (59.7%) continued treatment till last follow-up. A total of 33 (49.3%) were relapse-free. The median ARR decreased from one pre-treatment to zero post-treatment. Of 53 patients with complete EDSS data, the score improved or stabilized in 44 (83%; p < 0.05). Effectiveness was observed in aquaporin-4 (AQP4)-IgG (57.8% continued treatment, 46.7% relapse-free), MOG-IgG (3/5 continued treatment, 4/5 relapse-free), and seronegative NMOSD (64.7% continued treatment, 61.3% relapse-free). In 16 patients with associated steroids, 13 (81.2%) continued MMF till last follow-up versus 15 of 28 (53.6%) in the non-steroid group. Nine patients discontinued treatment for tolerability purpose.
  
  
  CONCLUSIONS
  MMF showed effectiveness and good tolerability as a first-line therapy in NMOSD, whatever the AQP4-IgG status. Concomitant use of oral steroids at start could limit the risk of treatment failure.
authors.affiliation: Service de Neurologie A and Eugène Devic EDMUS Foundation against Multiple Sclerosis, Observatoire Français de la Sclérose en Plaques (OFSEP), Hôpital Neurologique Pierre Wertheimer-GHE, Hospices Civils de Lyon, Bron, France/Department of Neurology, Hôpital Dupuytren, Limoges, France.
authors.forename: Alexis
authors.initials: A
authors.lastname: Montcuquet
authors.affiliation: Department of Neurology, and INSERM CIC-1434, CHU de Strasbourg, Strasbourg, France.
authors.forename: Nicolas
authors.initials: N
authors.lastname: Collongues
authors.affiliation: Department of Neurology, Hôpital Pitié-Salpêtrière, Paris, France.
authors.forename: Caroline
authors.initials: C
authors.lastname: Papeix
authors.affiliation: Clinique Neurologique, University of Lille, Lille, France.
authors.forename: Helene
authors.initials: H
authors.lastname: Zephir
authors.affiliation: Department of Neurology, Hôpital Timone, Marseille, France.
authors.forename: Bertrand
authors.initials: B
authors.lastname: Audoin
authors.affiliation: Department of Neurology, University Hospital of Nantes, Nantes, France.
authors.forename: David
authors.initials: D
authors.lastname: Laplaud
authors.affiliation: Department of Neurology, University Hospital of Rouen, Rouen, France.
authors.forename: Bertrand
authors.initials: B
authors.lastname: Bourre
authors.affiliation: Department of Neurology, and INSERM-CHU CIC-P 0005, CHU de Bordeaux, Bordeaux, France.
authors.forename: Bruno
authors.initials: B
authors.lastname: Brochet
authors.affiliation: Department of Neurology, University Hospital of Saint-Etienne, Saint-Etienne, France.
authors.forename: Jean-Philippe
authors.initials: JP
authors.lastname: Camdessanche
authors.affiliation: Department of Neurology, University Hospital of Montpellier, Montpellier, France.
authors.forename: Pierre
authors.initials: P
authors.lastname: Labauge
authors.affiliation: Department of Neurology, University Hospital of Dijon, Dijon, France.
authors.forename: Thibault
authors.initials: T
authors.lastname: Moreau
authors.affiliation: Department of Neurology, University Hospital of Purpan, Toulouse, France.
authors.forename: David
authors.initials: D
authors.lastname: Brassat
authors.affiliation: Department of Neurology, Hopital Tenon, Paris, France.
authors.forename: Bruno
authors.initials: B
authors.lastname: Stankoff
authors.affiliation: Department of Neurology and INSERM CIC-1434, CHU de Strasbourg, France.
authors.forename: Jerome
authors.initials: J
authors.lastname: de Seze
authors.affiliation: Service de Neurologie A and Eugène Devic EDMUS Foundation against Multiple Sclerosis, Observatoire Français de la Sclérose en Plaques (OFSEP), Hôpital Neurologique Pierre Wertheimer-GHE, Hospices Civils de Lyon, Bron, France/Lyon's Neuroscience Research Center, Team ONCOFLAM, Inserm U 1028/CNRS 5292, Lyon, France Université de Lyon, Université Claude Bernard Lyon 1, Lyon, France.
authors.forename: Sandra
authors.initials: S
authors.lastname: Vukusic
authors.affiliation: Service de Neurologie A and Eugène Devic EDMUS Foundation against Multiple Sclerosis, Observatoire Français de la Sclérose en Plaques (OFSEP), Hôpital Neurologique Pierre Wertheimer-GHE, Hospices Civils de Lyon, Bron, France/Lyon's Neuroscience Research Center, Team ONCOFLAM, Inserm U 1028/CNRS 5292, Lyon, France Université de Lyon, Université Claude Bernard Lyon 1, Lyon, France.
authors.forename: Romain
authors.initials: R
authors.lastname: Marignier
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D051401:Aquaporin 4 / Q000276:immunology; D001323:Autoantibodies / Q000276:immunology; D001324:Autoantigens / Q000276:immunology; D002648:Child; D015331:Cohort Studies; D005260:Female; D006801:Humans; D007074:Immunoglobulin G; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000627:therapeutic use*; D063308:Myelin-Oligodendrocyte Glycoprotein / Q000276:immunology; D009471:Neuromyelitis Optica / Q000188:drug therapy* / Q000276:immunology; D012008:Recurrence; D055815:Young Adult
chemical_list: C496030:AQP4 protein, human; D051401:Aquaporin 4; D001323:Autoantibodies; D001324:Autoantigens; D007074:Immunoglobulin G; D007166:Immunosuppressive Agents; C571326:MOG protein, human; D063308:Myelin-Oligodendrocyte Glycoprotein; D009173:Mycophenolic Acid
keywords: Aquaporin-4; myelin oligodendrocyte glycoprotein; neuromyelitis optica; treatment response