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

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

Author information

1
Department of Neurology, Medical School, Isfahan University of Medical Sciences, Isfahan, Iran; Isfahan Research Committee of Multiple Sclerosis (IRCOMS), Isfahan University of Medical Sciences, Isfahan, Iran.
2
Isfahan Research Committee of Multiple Sclerosis (IRCOMS), Isfahan University of Medical Sciences, Isfahan, Iran.
3
Isfahan Research Committee of Multiple Sclerosis (IRCOMS), Isfahan University of Medical Sciences, Isfahan, Iran; Medical Students' Research Committee, Isfahan University of Medical Sciences, Isfahan, Iran.
4
Nuffield Department of Clinical Neurosciences (Clinical Neurology), University of Oxford, John Radcliffe Hospital, Oxford, London, UK.
5
Department of Biological Sciences, California State University, Stanislaus, Turlock, CA, USA.
6
Isfahan Research Committee of Multiple Sclerosis (IRCOMS), Isfahan University of Medical Sciences, Isfahan, Iran; Medical Students' Research Committee, Isfahan University of Medical Sciences, Isfahan, Iran; Persia Research Center, Sady Hospital, Isfahan, Iran. Electronic address: mf.esfahani@yahoo.com.

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

PMID:
24139067
DOI:
10.1016/j.ejpn.2013.09.001
[Indexed for MEDLINE]
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