What is the role of immunomodulatory and cytotoxic therapies in the treatment of pediatric multiple sclerosis (MS)?

Updated: Jan 30, 2019
  • Author: Alice K Rutatangwa, DO, MSc; Chief Editor: Amy Kao, MD  more...
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A small number of children continue to have MS relapses despite treatment with the first-line DMT described above, although no consensus criteria exist for the definition of breakthrough disease in pediatric MS. In general, practitioners allow at least 6 months of observation on a given treatment prior to deeming the treatment suboptimal.

Immunomodulatory and cytotoxic agents that are used in adult MS in the event of suboptimal response to interferon-beta and/or glatiramer acetate have been used in a small number of pediatric patients. There are no reports on the use of fingolimod in pediatric MS.

Natalizumab appears well tolerated by children with MS in whom first-line therapies have failed. Patients experience less clinical relapses, and MRI scans show no enhancing lesions. [52] There have been no reports to date of PML in association with natalizumab use in children. [53]

There have been no published reports of mitoxantrone use in the pediatric population except for a few cases followed at the US Pediatric MS Centers of Excellence. [54] The authors suggest caution with its use given the reported significant side effects of leukemia and cardiomyopathy.

In the pediatric MS population, only one case report of rituximab has been published. [55]

There is limited evidence to support the use of cyclophosphamide in children. Caution should be exercised given the risks of infertility and secondary neoplasm.

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