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.


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.
Jacob
Anu
A
Department of Neurology, Mayo Clinic College of Medicine, Rochester, Minnesota 55905, USA.
Matiello
Marcelo
M
Weinshenker
Brian G
BG
Wingerchuk
Dean M
DM
Lucchinetti
Claudia
C
Shuster
Elizabeth
E
Carter
Jonathan
J
Keegan
B Mark
BM
Kantarci
Orhun H
OH
Pittock
Sean J
SJ
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
C496030:AQP4 protein, human; D000894:Anti-Inflammatory Agents, Non-Steroidal; D051401:Aquaporin 4; D007074:Immunoglobulin G; D009173:Mycophenolic Acid