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Treatment of pediatric multiple sclerosis and variants

D. Pohl, MD, E. Waubant, MD, PhD, B. Banwell, MD, D. Chabas, MD, PhD, T. Chitnis, MD, B. Weinstock-Guttman, MD, S. Tenembaum, MD for the International Pediatric MS Study Group

From the Department of Pediatrics and Pediatric Neurology (D.P.), Georg-August-University Goettingen, Germany; Multiple Sclerosis Center at UCSF (E.W., D.C.), San Francisco, CA; Department of Pediatric Neurology (B.B.), The Hospital for Sick Children, University of Toronto, Canada; Massachusetts General Hospital for Children (T.C.), Brigham & Women’s Hospital, Harvard Medical, Boston; Baird MS Center (B.W.-G.), Jacobs Neurological Institute, Buffalo, NY; and Department of Pediatric Neurology (S.T.), National Pediatric Hospital, Dr. J. P. Garrahan, Buenos Aires, Argentina.

Address correspondence and reprint requests to Dr. Emmanuelle Waubant, UCSF MS Center, 350 Parnassus Street, Suite 908, San Francisco, CA 94117; e-mail: Emmanuelle.Waubant{at}ucsf.edu

Studies in adult patients with multiple sclerosis (MS) suggest significant benefit of early treatment initiation. However, there are no approved therapies for children and adolescents with MS. For adult MS, tolerability and efficacy of several immunomodulatory and immunosuppressive drugs have been demonstrated. Guidelines for the use of these MS therapies in children do not exist. Several small cohort studies of the safety and tolerability of disease-modifying therapies (DMT) in children and adolescents with MS have been recently reported. The side effects of interferon beta (IFNB) and glatiramer acetate (GA) appear to be similar to those reported by adults. The long-term tolerability and safety have yet to be established and efficacy data have yet to be studied. In view of the potential for significant long-term physical and cognitive disability in children with MS, and recent evidence that initiation of immunomodulatory therapy early in the course of MS improves long-term prognosis, an increasing number of children and adolescents with MS are being offered the DMT approved for adults. This review summarizes current knowledge of DMT in pediatric MS and experience in several centers treating pediatric MS and MS variants such as neuromyelitis optica or Devic disease, Balo concentric sclerosis, Marburg acute MS, and Schilder disease (myelinoclastic diffuse sclerosis). Finally, an overview of symptomatic MS therapies and experiences with these treatments in pediatric patients is provided.

*The first authors contributed equally to this work.

Members of the International Pediatric MS Study Group are listed in the Appendix.

Disclosure: The authors report no conflicts of interest.

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