Efficacy and Safety of Mitoxantrone in Patients With Highly Relapsing Neuromyelitis Optica

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.