Affiliations: Department of General Pediatrics, University
Children's Hospital, Duesseldorf, Germany | Department of Neurology, University Children's
Hospital, Duesseldorf, Germany | Department of Diagnostic Radiology, University
Children's Hospital, Duesseldorf, Germany
Note: [] Correspondence: Dr. Martina Heckel, Department of General
Pediatrics, University Children's Hospital, Heinrich-Heine-University
Düsseldorf Moorenstr. 5, D-40225 Düsseldorf, Germany. Tel.: +49 211
811 7687; Fax: +49 211 811 8757; E-mail: martinaheckel@web.de
Abstract: 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.
