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Safety and efficacy of mitoxantrone in pediatric patients with aggressive multiple sclerosis
Masoud Etemadifar1, Parisa Afzali2, Seyed-Hossein Abtahi3
1Department 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.
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.
Insights
Mitoxantrone (MX) shows promise for treating pediatric aggressive multiple sclerosis (MS), with most patients experiencing reduced relapses and improved MRI findings. Adverse events were generally mild and transient, warranting further research.
Area of Science:
- Pediatric Neurology
- Neuroimmunology
- Clinical Pharmacology
Background:
- Multiple Sclerosis (MS) in pediatric patients presents unique challenges.
- Aggressive forms of MS require effective therapeutic interventions.
Purpose of the Study:
- To evaluate the safety and efficacy of mitoxantrone (MX) in children with aggressive multiple sclerosis (MS).
Main Methods:
- Retrospective analysis of pediatric MS patients treated with mitoxantrone.
- Assessment of demographic, clinical, and magnetic resonance imaging (MRI) parameters.
Main Results:
- 73% of patients were relapse-free after a median 30-month follow-up.
- Significant improvements observed in Expanded Disability Status Scale (EDSS) scores and reduced gadolinium-enhancing MRI lesions.
- Reported adverse events, including nausea, fatigue, and alopecia, were mild and transient.
Conclusions:
- Mitoxantrone appears to be a viable treatment option for pediatric patients with worsening relapsing-remitting MS (RRMS) and secondary progressive MS (SPMS).
- Careful patient selection, administration protocols, and close monitoring for adverse events are recommended.
- Further multinational research is necessary to confirm efficacy and safety.
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