Safety and efficacy of mitoxantrone in pediatric patients with aggressive multiple sclerosis

Eur J Paediatr Neurol. 2014 Mar;18(2):119-25. doi: 10.1016/j.ejpn.2013.09.001. Epub 2013 Oct 7.

Abstract

Objective: The purpose of this study was to assess the safety and efficacy of mitoxantrone (MX) in pediatric patients with aggressive multiple sclerosis (MS).

Methods: A retrospective analysis on pediatric MS patients treated with MX was performed with regards to demographic/clinical parameters and magnetic resonance imaging (MRI) findings.

Results: 19 definite pediatric MS cases with mean ± SD age of 15.4 ± 2.8 years underwent 20 mg MX for control of their severe/frequent relapses, high EDSS score or new and active brain MRI lesions. After a median [IQR] follow-up period of 30[12-60] months, 14 cases (73%) were relapse free; the EDSS score decreased by at least 0.5 in 16 cases (84.2%); and gadolinium-enhancing lesion volume fell by 84.2% in 16 cases. Adverse events included nausea and vomiting, fatigue, alopecia, palpitation, cardiomyopathy and mild leukopenia. All adverse events were mild and transient.

Conclusion: Our results suggest MX is a good candidate for treatment of children with worsening RRMS and SPMS. Recommendations regarding patient selection, treatment administration, and close follow-up should be considered. Continuing research is needed to establish its efficacy and safety profile in a multinational collaboration with careful follow-up of adverse events.

Keywords: Aggressive; Mitoxantrone; Multiple sclerosis; Pediatric.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Child
  • Female
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Mitoxantrone / therapeutic use*
  • Multiple Sclerosis, Chronic Progressive / drug therapy*
  • Multiple Sclerosis, Relapsing-Remitting / drug therapy*
  • Retrospective Studies
  • Young Adult

Substances

  • Immunosuppressive Agents
  • Mitoxantrone