Abstract

Background: Severe side effects such as cardiotoxicity and leukemia limit long-term use of mitoxantrone (MTX) despite its recommendation in patients with malignant forms of relapsing remitting (RR) and secondary progressive (SP) multiple sclerosis (MS). Methods: We analyzed data on tolerability, measured as compliance with the treatment, and patients’ acceptance of an alternative MTX treatment schedule in 12 patients with aggressive RRMS or SPMS treated for at least 3 years with low/delayed dose MTX. Results: Twelve patients received 9–17 cycles of MTX treatment, with individual median doses of 7.6–12.16 mg/m<sup>2</sup>/course during 36 to 114 (median 49.5) months resulting in cumulative doses of 101.9–143.0 mg/m<sup>2</sup> per patient. The overall follow-up period was 37–122 (median 69.5) months. Treatment was well tolerated and appreciated by patients according to the Treatment Satisfaction-Visual Analogue Scale. No significant safety findings were seen concerning cardiological, hematological and oncological follow-up. The patients showed a stabilization of disease progression and a reduced annual relapse rate. Conclusions: Delayed, and/or dose-reduced, long-term MTX regimens over several years might represent a feasible alternative treatment for MS patients without any other therapeutic options.

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