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

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/m ², 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.