Etemadifar M, Afzali P, Abtahi SH, Ramagopalan SV, Nourian SM, Murray RT, Fereidan-Esfahani M. Safety and efficacy of mitoxantrone in paediatric patients with aggressive multiple sclerosis. Eur J Paediatr Neurol. 2013 Oct. doi:pii: S1090-3798(13)00131-1. 10.1016/j.ejpn.2013.09.001. [Epub ahead of print]
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 paediatric MS patients treated with MX was performed with regards to demographic/clinical parameters and magnetic resonance imaging (MRI) findings.
RESULTS: 19 definite paediatric 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 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.
It will be interesting to look at the long-term follow up