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Neuropediatrics 2011; 42(1): 7-12
DOI: 10.1055/s-0031-1275345
Original Article

© Georg Thieme Verlag KG Stuttgart · New York

Long-Term Follow-Up of Pediatric Patients Treated with Mitoxantrone for Multiple Sclerosis

B. Kornek1 , G. Bernert2 , K. Rostasy3 , E. Mlczoch4 , M. Feucht5 , D. Prayer6 , K. Vass1 , R. Seidl7
  • 1Department of Neurology, Medical University of Vienna, Vienna, Austria
  • 2Gottfried v. Preyer's Hospital for Sick Children, Vienna, Austria
  • 3Department of Pediatrics IV, Division of Neuropediatrics and Inborn Errors of Metabolism, Medical University of Innsbruck, Innsbruck, Austria
  • 4Department of Paediatrics and Adolescent Medicine, Division of Paediatric Cardiology, Medical University of Vienna, Vienna, Austria
  • 5Department of Paediatrics and Adolescent Medicine, Division of Neonatology, Intensive Care and Neuropaediatrics, Medical University of Vienna, Vienna, Austria
  • 6Department of Radiology, Division of Neuroradiology, Medical University of Vienna, Vienna, Austria
  • 7Department of Paediatrics and Adolescent Medicine, Division of Pulmology, Allergology and Endocrinology, Medical University of Vienna, Vienna, Austria
Further Information

Publication History

received 27.08.2010

accepted 7.03.2011

Publication Date:
09 May 2011 (online)

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Abstract

The chemotherapeutic agent mitoxantrone is approved for the treatment of aggressive multiple sclerosis (MS) in adults. Its use, however, is limited by the risk of severe adverse events including cardiotoxicity, myelosuppression, liver toxicity and secondary leukemia. The aim of this retrospective study is to present data on the safety, tolerability and efficacy of mitoxantrone in a small cohort of children with MS.

4 pediatric MS patients with a high relapse rate or severe, disabling relapses were treated with mitoxantrone and followed for 3.8–18 years. The cumulative dose of mitoxantrone was 36, 68, 84 and 120 mg/m2, respectively. The frequency and severity of relapses as well as disability scores, decreased in the year after treatment onset. Short-term adverse events were transient in all cases. Cardiac monitoring by transthoracic echocardiography (TTE) showed asymptomatic left ventricular dysfunction during treatment in 1 patient, which was again normal at the next assessment. Long-term adverse events, including late-onset mitoxantrone-induced cardiotoxicity or secondary leukemia did not occur during the follow-up period.

In our cohort of pediatric MS patients, mitoxantrone showed short-term reduction of disease activity and no long-term adverse events on follow-up. However, the risk of mitoxantrone-induced cardiotoxicity or toxic leukemia remains a life-long threat.

Key words

multiple sclerosis - children - mitoxantrone

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Correspondence

Barbara KornekMD 

Department of Neurology

Medical University of Vienna

Währinger Gürtel 18– 20

A–1090 Vienna

Austria

Phone: +43/1/40400 3145

Fax: +43/1/40400 6215

Email: barbara.bajer-kornek@meduniwien.ac.at

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