Mitoxantrone in NMO Spectrum Disorder in a Large Multicenter Cohort in French Caribbean

BACKGROUND AND OBJECTIVES: Preventing relapses in neuromyelitis Optica spectrum disorder (NMOSD) is a primary goal. New effective molecules are often expensive and not readily available in regions with fragile health systems. Assessing the efficacy and safety of less costly therapeutic alternatives is necessary. We aim to evaluate the efficacy and safety of mitoxantrone (MiTX) in NMOSD. METHODS: This is an observational, multicenter, open study of 86 NMOSD-treated patients with prospective follow-up over 30 years. The first endpoint was the first relapse at the 96-week follow-up. The secondary endpoints were to evaluate the median delay to relapse, the annualized relapse rate (ARR), and the Expanded Disability Status Scale (EDSS) at 96 weeks of follow-up and to assess risk factors of relapse and the occurrence of severe adverse effects. RESULTS: At 96-week follow-up, 71% of our patients were relapse-free, and it was 87% when patients were treated with MiTX from the first attack. The ARR dropped from 0.85 (±0.55) to 0.32 (±0.63) (p < 0.001) and EDSS from 4.9 (±2.4) to 4.2 (±2.6) (p < 0.001). AQP4-IgG seropositivity (hazard ratio [HR] 12.3, 95% CI 1.64–91.6, p = 0.015), a delay between the first attack and MiTX ≥24 months (HR 2.76, 95% CI 1.23–6.17, p = 0.014), and a pretreatment ARR ≥1 (HR 2.38, 95% CI 1.05–5.39, p = 0.037) were predictors of relapse. During the entire follow-up, severe secondary adverse events occurred in 3 patients (3.5%). DISCUSSION: MiTX is an effective and safe treatment for most of our patients, drastically less expensive than new molecules, and could be allowed in NMOSD Afro-descendant patients in geographical areas where access to care is difficult.