Effectiveness of mycophenolate mofetil as first-line therapy in AQP4-IgG, MOG-IgG, and seronegative neuromyelitis optica spectrum disorders.
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.


CONCLUSION
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.
['Montcuquet|Alexis|A|', 'Collongues|Nicolas|N|', 'Papeix|Caroline|C|', 'Zephir|Helene|H|', 'Audoin|Bertrand|B|', 'Laplaud|David|D|', 'Bourre|Bertrand|B|', 'Brochet|Bruno|B|', 'Camdessanche|Jean-Philippe|JP|', 'Labauge|Pierre|P|', 'Moreau|Thibault|T|', 'Brassat|David|D|', 'Stankoff|Bruno|B|', 'de Seze|Jerome|J|', 'Vukusic|Sandra|S|', 'Marignier|Romain|R|', '|||']
D000293:Adolescent D000328:Adult D000368:Aged D051401:Aquaporin 4 D001323:Autoantibodies D001324:Autoantigens D002648:Child D015331:Cohort Studies D005260:Female D006801:Humans D007074:Immunoglobulin G D007166:Immunosuppressive Agents D008297:Male D008875:Middle Aged D009173:Mycophenolic Acid D063308:Myelin-Oligodendrocyte Glycoprotein D009471:Neuromyelitis Optica D012008:Recurrence D055815:Young Adult
neuromyelitis optica myelin oligodendrocyte glycoprotein Aquaporin-4 treatment response