Which Treatment Is Best for Neuromyelitis Optica?

Summary and Comment |
February 4, 2014

Which Treatment Is Best for Neuromyelitis Optica?

  1. Robert T. Naismith, MD

A retrospective analysis suggests benefits from all three treatments, with subtle differences.

  1. Robert T. Naismith, MD

Neuromyelitis optica (NMO) has been shown to be a relapsing disease with a high risk of disability that benefits from long-term immunosuppression (NEJM JW Neurol May 29 2012). However, phase III therapeutic trials are lacking for this rare disease. Common options include azathioprine with at least 6 months of prednisone, mycophenolate mofetile with at least 6 months of prednisone, and rituximab. The present study is a retrospective comparison of these three regimens at two academic centers with large NMO populations.

Of 90 patients with NMO, 32 were treated with azathioprine, 28 with mycophenolate, and 30 with rituximab. Comparing pretreatment with post-treatment relapses, azathioprine reduced the annualized relapse rate by 72% (from 2.26 to 0.63), mycophenolate by 87% (from 2.61 to 0.33), and rituximab by 89% (from 2.89 to 0.33). Efficacy improved when each agent was dosed optimally. Freedom from relapses was achieved in 47% of patients with azathioprine, 64% with mycophenolate, and 67% with rituximab.


This study is valuable because it provides head-to-head comparative data among the neuromyelitis optica treatments. The findings emphasize that each of the treatments works and must be dosed optimally. Optimal means following lymphocyte counts with azathioprine and mycophenolate, using prednisone for at least the first 6 months with azathioprine and mycophenolate, and retreating on schedule with rituximab to maintain B-cell depletion (NEJM JW Neurol Sep 13 2011). Many patients have a favorable treatment response to azathioprine, but there are clear nonresponders as well. Mycophenolate may have an edge over azathioprine in efficacy, relative ease of use (i.e., no need for thiopurine methyltransferase gene determination), and dosing, but mycophenolate has significant teratogenicity, a concern in treating women of childbearing potential. Rituximab has advantages for efficacy, infrequent dosing, and relatively rapid onset. Rituximab may be preferential for severe, rapidly relapsing NMO, whereas the two oral agents can be considered based on clinician familiarity and family planning considerations.

Editor Disclosures at Time of Publication

  • Disclosures for Robert T. Naismith, MD at time of publication Consultant / Advisory board Acorda Therapeutics; Biogen Idec; EMD Serono; Genzyme; Questcor Speaker's bureau Acorda Therapeutics; Bayer Healthcare; Biogen Idec; Genzyme Grant / research support NIH; Acorda Therapeutics; National Multiple Sclerosis Society Leadership positions in professional societies Consortium of MS Centers (Speaker); National Multiple Sclerosis Society (Local Chair of Clinical Advisory Committee; Program Services Committee, Research Advocate, and Research Champion)


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