Mitoxantrone as rescue therapy in two children with worsening multiple sclerosis

Multiple sclerosis (MS) is an inflammatory, demyelinating disease of the central nervous system of unknown etiology. In the acute phase, MS is treated with corticosteroids, whereas drugs, such as interferon-beta and glatiramer acetate, are currently used as continuous immunomodulatory therapies. Recently, the cytostatic drug mitoxantrone was reported to be well tolerated and therapeutically effective in deteriorating MS in adults. Under the concept of an escalating therapy regimen, we addressed if mitoxantrone could decrease the frequency of relapses and stabilize the course of the disease not only in adults but also in children. Two adolescents (female 13 yrs.; male 14 yrs.) with deteriorating relapsing-remitting MS were selected after various immunomodulatory treatments had failed before. After informed consent had been obtained from the parents and the patients, we administered mitoxantrone intravenously in three-month intervals for 3.5 years and 12 months, respectively. Therapy with mitoxantrone was well tolerated in both patients. In the female patient a decrease in the number of relapses, and an improvement in clinical disability as measured by the Expanded Disability Status Scale was observed. In the male patient, a stabilization of the clinical course was achieved. We conclude that treatment with mitoxantrone could be considered in children with deteriorating MS when other therapeutic agents have failed. However, controlled studies in the pediatric age group are needed in order to assess the benefit of this chemotherapeutic agent and to rule out long-term adverse effects.