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Original Contribution
July 2006

Study of Mitoxantrone for the Treatment of Recurrent Neuromyelitis Optica (Devic Disease)

Author Affiliations

Author Affiliations: The William C. Baird Multiple Sclerosis Center, Jacobs Neurological Institute (Drs Weinstock-Guttman, Ramanathan, and Lincoff and Ms Feichter) and Department of Pharmaceutical Sciences (Dr Ramanathan), State University of New York, Buffalo; and Partners Multiple Sclerosis Center, Center for Neurological Imaging, Departments of Neurology and Radiology, Brigham and Women's Hospital, Harvard Medical School, Boston, Mass (Drs Napoli, Sharma, and Bakshi).

Arch Neurol. 2006;63(7):957-963. 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  clinicaltrials.gov Identifier: NCT00304291