Study of mitoxantrone for the treatment of recurrent neuromyelitis optica (Devic disease)

Arch Neurol. 2006 Jul;63(7):957-63. doi: 10.1001/archneur.63.7.957.


Background: Neuromyelitis optica is a severe demyelinating disease that selectively involves the optic nerves and the spinal cord but usually spares the brain. It is considered to have a B-cell-induced pathogenesis. Mitoxantrone hydrochloride, a synthetic anthracenedione approved for worsening relapsing-remitting multiple sclerosis and secondary progressive multiple sclerosis, has been shown to primarily suppress the humoral response.

Objective: To evaluate the benefit of mitoxantrone treatment in patients with relapsing neuromyelitis optica.

Design: Prospective 2-year study.

Setting: Academic multiple sclerosis center.

Patients: Five patients (3 women and 2 men) with an age range of 20 to 51 years and an Expanded Disability Status Scale score of 2.5 to 6.5 (mean +/- SD, 4.40 +/- 1.88).

Interventions: Monthly intravenous infusions of mitoxantrone hydrochloride, 12 mg/m2, for 6 months followed by 3 additional treatments every 3 months.

Main outcome measures: Expanded Disability Status Scale score measured every 3 months and during relapses; findings on orbital, brain, and spinal cord magnetic resonance images performed at baseline and at 3, 6, 12, 18, and 24 months; and visual evoked potentials and results of ophthalmologic evaluations performed at baseline and annually.

Results: During the 2 years of treatment, 2 patients each had a relapse once within the initial 5 months of treatment (1 severe and 1 moderate). Improvement was seen clinically and on magnetic resonance images in 4 patients. Patients generally tolerated the treatment well, although 1 patient had a reversible decrease in cardiac ejection fraction.

Conclusion: Our results suggest a beneficial effect of mitoxantrone treatment for relapsing neuromyelitis optica.

Trial registration: NCT00304291.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Antineoplastic Agents / administration & dosage*
  • Antineoplastic Agents / adverse effects
  • Central Nervous System / pathology
  • Disability Evaluation
  • Female
  • Humans
  • Injections, Intravenous
  • Magnetic Resonance Imaging
  • Male
  • Middle Aged
  • Mitoxantrone / administration & dosage*
  • Mitoxantrone / adverse effects
  • Neuromyelitis Optica / drug therapy*
  • Neuromyelitis Optica / pathology
  • Prospective Studies
  • Recurrence
  • Treatment Outcome


  • Antineoplastic Agents
  • Mitoxantrone

Associated data