Analysis of the treatment of neuromyelitis optica.
BACKGROUND
Treatment options for neuromyelitis optica (NMO) are currently based on small retrospective case series and open label studies, ranging from 10 to 103 patients.


OBJECTIVE
To compare the efficacy and tolerability of azathioprine, cyclophosphamide, mycophenolate, and rituximab in patients with neuromyelitis optica.


METHODS
This is a retrospective chart review and telephone follow-up study of 71 patients with NMO or NMO spectrum disorder, 54 of whom were treated with the study drugs. We compared adverse events, annualized relapse rates and expanded disability status scales before and after treatment.


RESULTS
The median ARR decreased from 1.17 to 0.25 on rituximab (P<0.01), 0.92 to 0.56 on azathioprine (P=0.475), 1.06 to 0.39 on mycophenolate (P<0.05) and 1.30 to 0.92 on cyclophosphamide (P=0.746). When compared directly to azathioprine, rituximab significantly reduced relapse rates (P=0.021). The median EDSS decreased from 7 to 5 on rituximab (P<0.01) and 7 to 6 on azathioprine (P<0.01), and did not change significantly on mycophenolate (4 to 5; P=0.463) or cyclophosphamide (6.5 to 6.5; P=0.881). Twenty-five percent of patients noted adverse events on rituximab, 36% on azathioprine, 36% on mycophenolate, and 80% on cyclophosphamide.


CONCLUSIONS
Rituximab significantly reduces relapse rates and improves disability while maintaining comparable tolerability to other immunosuppressive treatments for NMO.
Department of Neurology, New York University Langone Medical Center, USA. Electronic address: jose.torres2@nyumc.org.
Jose
J
Torres
Department of Neurology, University of Pennsylvania, USA.
Amy
A
Pruitt
Department of Neurology, New York University Langone Medical Center, USA.
Laura
L
Balcer
Department of Neurology, New York University Langone Medical Center, USA.
Steven
S
Galetta
Department of Neurology, University of Pennsylvania, USA.
Clyde
C
Markowitz
Department of Neurology, University of Pennsylvania, USA.
Nabila
N
Dahodwala
D000293:Adolescent; D000328:Adult; D000368:Aged; D001379:Azathioprine / Q000494:pharmacology*; D002648:Child; D003520:Cyclophosphamide / Q000494:pharmacology*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007155:Immunologic Factors / Q000494:pharmacology*; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000494:pharmacology*; D009471:Neuromyelitis Optica / Q000188:drug therapy*; D017063:Outcome Assessment, Health Care; D012008:Recurrence; D012189:Retrospective Studies; D000069283:Rituximab / Q000494:pharmacology*; D055815:Young Adult
D007155:Immunologic Factors; D000069283:Rituximab; D003520:Cyclophosphamide; D009173:Mycophenolic Acid; D001379:Azathioprine
Azathioprine; Devic's syndrome; Mycophenolate; NMO; Neuromyelitis optica; Rituximab; Treatment