Mycophenolate mofetil in the treatment of neuromyelitis optica spectrum disorder

So Young Huh, Su Hyun Kim, Jae Won Hyun, Ae Ran Joung, Min Su Park, Byung Jo Kim, Ho Jin Kim

Research output: Contribution to journalArticle

49 Citations (Scopus)

Abstract

IMPORTANCE: Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune inflammatory disorder of the central nervous system. Recently, various immunosuppressant medications were introduced as therapeutic options for preventing relapse of NMOSD. However, our understanding of the effectiveness ofmycophenolate mofetil (MMF) in treating patients with NMOSD is based on only a small number of studies. OBJECTIVE: To evaluate the efficacy and safety of MMF treatment in patients with NMOSD. DESIGN, SETTING, AND PARTICIPANTS: A 3-center retrospective review of our experiences, examining results from 59 patients with NMOSD (24 with neuromyelitis optica and 35 with a limited form of the disease) who were treated with MMF (1000-2000mg/d). MAIN OUTCOMES AND MEASURES: Patients' annualized relapse rate, disability as measured by the Expanded Disability Status Scale, and experience of adverse effects due to MMF were assessed. RESULTS: Of the 59 patients, 1 with NMOSD who had to discontinue MMF use in the first month due to a rash was excluded. The remaining 58 patients were included in the drug-efficacy analysis. The median post-MMF annualized relapse rate was significantly lower than the pre-MMF annualized relapse rate (0.0 vs 1.5; P < .001). The Expanded Disability Status Scale scores also significantly decreased after MMF treatment (3.0 vs 2.5; P = .005). Thirty-five patients (60%) were relapse free, and Expanded Disability Status Scale scores were stabilized or improved in 53 patients (91%). Fourteen patients discontinued MMF treatment owing to ongoing relapse (10 patients), rash (1 patient), pregnancy (1 patient), and financial problems (2 patients), but MMF was generally well tolerated. CONCLUSIONS AND RELEVANCE: In this observational study, MMF treatment induced reduction of relapse frequency, stabilized or improved disability, and was well tolerated in patients with NMOSD.

Original languageEnglish
Pages (from-to)1372-1378
Number of pages7
JournalJAMA Neurology
Volume71
Issue number11
DOIs
Publication statusPublished - 2014 Jan 1

    Fingerprint

ASJC Scopus subject areas

  • Arts and Humanities (miscellaneous)
  • Clinical Neurology
  • Medicine(all)

Cite this

Huh, S. Y., Kim, S. H., Hyun, J. W., Joung, A. R., Park, M. S., Kim, B. J., & Kim, H. J. (2014). Mycophenolate mofetil in the treatment of neuromyelitis optica spectrum disorder. JAMA Neurology, 71(11), 1372-1378. https://doi.org/10.1001/jamaneurol.2014.2057