Is there a new place for mitoxantrone in the treatment of multiple sclerosis?

AIM OF THE STUDY: To compare the clinical and neuroradiological efficacy of mitoxantrone (MTX) in various forms of multiple sclerosis (MS), to ascertain whether there is a new place for the drug in the treatment regimen of the disease, as well as to determine its safety profile. CLINICAL RATIONALE FOR THE STUDY: Due to the increasing availability of new immunomodulatory therapies in multiple sclerosis (MS), there is a strong need to re-identify clinical variants and stages of the disease in which mitoxantrone (MTX) can be the most effective form of treatment. MATERIALS AND METHODS: This was a retrospective, non-randomised, observational study evaluating a cohort of 100 MS patients (36 relapsing-remitting - RRMS, 36 secondary progressive - SPMS, and 28 primary progressive - PPMS). 59% of the RRMS patients had discontinued immunomodulatory therapies (IMTs) within the two years preceding MTX infusion. Patients' disability levels, based on the Kurtzke Expanded Disability Status Scale (EDSS) as well as haematological and echocardiographic parameters, were assessed at baseline and before every infusion. Magnetic resonance imaging (MRI) were performed at entry and after termination of treatment. RESULTS: We observed a decrease in the median EDSS score from 4.0 at baseline to 3.5 at the end of MTX infusion in the RRMS subgroup, an increase from 4.5 to 5.25 in the PPMS subgroup, and a stable value of 5 points in the SPMS subgroup (p < 0.0001). During the treatment period, 97% of patients with initial RRMS were free of exacerbations. The baseline EDSS in the RRMS subgroup, as well as the ineffectiveness of previous IMTs, suggested the beginning of conversion to SPMS. We found an 86% decrease in the proportion of patients with gadolinium-enhancing lesions on MRI after MTX infusions. There were no lifethreatening adverse events of MTX during the period of evaluation. CONCLUSIONS AND CLINICAL IMPLICATIONS: Mitoxantrone can be considered as a valuable therapeutic option for patients who are on the borderline of RRMS and SPMS.