Efficacy and safety of mitoxantrone in patients with highly relapsing neuromyelitis optica

Arch Neurol. 2011 Apr;68(4):473-9. doi: 10.1001/archneurol.2010.322. Epub 2010 Dec 13.


Objective: To evaluate the efficacy and safety of mitoxantrone hydrochloride and determine how it exhibits a differential inhibitory effect on subsets of B cells in patients with highly relapsing neuromyelitis optica (NMO).

Design: Retrospective case series with prospective follow-up.

Setting: Three referral medical centers in the Republic of Korea.

Patients: Twenty patients with highly relapsing NMO or neuromyelitis optica spectrum disorder who had at least 2 relapses during the year preceding the start of mitoxantrone treatment, despite other immunotherapies.

Intervention: Infusions of mitoxantrone up to a maximum cumulative dose of 120 mg/m(2).

Main outcome measures: Annualized relapse rate, disability according to the Expanded Disability Status Scale score, and fraction of CD27(+)CD19(+) memory B cells.

Results: During mitoxantrone treatment, the median annualized relapse rate was reduced by 75%, and 50% of patients became relapse free. Disability improved or stabilized in all patients. No patients had serious adverse effects during the mean follow-up period of 41 months after completing therapy. Flow cytometric analysis of cell surface markers revealed that mitoxantrone treatment preferentially affected CD27(+)CD19(+) memory B cells.

Conclusions: Treatment with mitoxantrone in patients with highly relapsing NMO significantly reduces relapse rates, resulting in subsequent functional stabilization or improvement.

Publication types

  • Comparative Study
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Female
  • Follow-Up Studies
  • Humans
  • Infusions, Intravenous
  • Leukopenia / chemically induced
  • Leukopenia / immunology
  • Male
  • Middle Aged
  • Mitoxantrone / administration & dosage*
  • Mitoxantrone / adverse effects*
  • Nausea / chemically induced
  • Nausea / immunology
  • Neuromyelitis Optica / drug therapy*
  • Neuromyelitis Optica / immunology
  • Neuromyelitis Optica / prevention & control
  • Prospective Studies
  • Retrospective Studies
  • Secondary Prevention
  • Treatment Outcome
  • Young Adult


  • Mitoxantrone