title: Safety and efficacy of mitoxantrone in pediatric patients with aggressive multiple sclerosis.
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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: 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.
authors.forename: Masoud
authors.initials: M
authors.lastname: Etemadifar
authors.affiliation: Isfahan Research Committee of Multiple Sclerosis (IRCOMS), Isfahan University of Medical Sciences, Isfahan, Iran.
authors.forename: Parisa
authors.initials: P
authors.lastname: Afzali
authors.affiliation: 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.
authors.forename: Seyed-Hossein
authors.initials: SH
authors.lastname: Abtahi
authors.affiliation: Nuffield Department of Clinical Neurosciences (Clinical Neurology), University of Oxford, John Radcliffe Hospital, Oxford, London, UK.
authors.forename: Sreeram V
authors.initials: SV
authors.lastname: Ramagopalan
authors.affiliation: Department of Biological Sciences, California State University, Stanislaus, Turlock, CA, USA.
authors.forename: Sayed-Mohammadamin
authors.initials: SM
authors.lastname: Nourian
authors.affiliation: Isfahan Research Committee of Multiple Sclerosis (IRCOMS), Isfahan University of Medical Sciences, Isfahan, Iran.
authors.forename: Richard T
authors.initials: RT
authors.lastname: Murray
authors.affiliation: 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.
authors.forename: Mahboobeh
authors.initials: M
authors.lastname: Fereidan-Esfahani
mesh_terms: D000293:Adolescent; D002648:Child; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008942:Mitoxantrone / Q000627:therapeutic use*; D020528:Multiple Sclerosis, Chronic Progressive / Q000188:drug therapy*; D020529:Multiple Sclerosis, Relapsing-Remitting / Q000188:drug therapy*; D012189:Retrospective Studies; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D008942:Mitoxantrone
keywords: Aggressive; Mitoxantrone; Multiple sclerosis; Pediatric