Mitoxantrone in worsening secondary progressive multiple sclerosis: A prospective, open-label study

Area of Science:

• Neurology
• Immunology
• Pharmacology

Background:

• Mitoxantrone (MX) is an antineoplastic agent used for multiple sclerosis (MS).
• Limited data exists on the efficacy of intravenous (IV) MX in patients with secondary progressive MS (SPMS).

Purpose of the Study:

• To monitor the effects of IV MX in SPMS patients with worsening conditions despite methylprednisolone treatment.
• To assess the efficacy and tolerability of IV MX in a diverse SPMS patient group.

Main Methods:

• Prospective, open-label study of 48 SPMS patients receiving IV MX (12 mg/m(2) every 12 weeks) for up to 2 years.
• Efficacy assessed via Extended Disability Status Scale (EDSS); tolerability monitored through examinations and lab tests.
• Cardiac function evaluated using multigated acquisition scanning every 24 weeks.

Main Results:

• No significant change in mean EDSS score observed post-treatment or at 1-year follow-up.
• 14.6% of patients experienced severe adverse effects, including leukopenia and septicemia.
• Two patients had asymptomatic decreases in cardiac ejection fraction, leading to treatment discontinuation.

Conclusions:

• IV MX treatment for up to 2 years showed no significant change in EDSS scores for worsening SPMS patients.
• The study highlights potential adverse events and limited efficacy in this patient cohort.