Clinical Medicine Insights: Therapeutics

Background and purpose: Mitoxantrone (MX) (Novantrone®) is approved in Canada for certain refractory cancers and acute non-lymphocytic leukemias. It has FDA approval as an immunomodulatory agent for use in secondary progressive multiple sclerosis (SPMS). The general aim of this study is to evaluate the efficacy, safety, and tolerability of MX in SPMS.

Experimental approach: A single-centre, open-label, non-randomized study was conducted in patients with a 6 month history of SPMS. The primary parameters used to assess efficacy and safety were EDSS scores and the multiple-gated acquisition scan (MUGA) scores, respectively.

Key results: The MX-treatment group experienced a high dropout rate due to significantly reduced ventricular ejection fraction. EDSS scores from baseline to follow-up revealed no statistical difference between active control and MX-treatment groups.

Conclusion: MX treatment may not slow disease progression in certain forms of SPMS and may be associated with a high risk of cardiotoxicity.