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Paediatr Drugs. 2019 Jun;21(3):137-152. doi: 10.1007/s40272-019-00338-6.

Improving Outcomes in Pediatric Multiple Sclerosis: Current and Emerging Treatments.

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Department of Pediatrics, Faculty of Medicine and Dentistry, Women and Children's Health Research Institute, University of Alberta, Edmonton, AB, Canada.
Division of Neurology, Department of Pediatrics, The Hospital for Sick Children, 555 University Ave, Toronto, ON, M5G 1X8, Canada.
Division of Neurosciences and Mental Health, SickKids Research Institute, Toronto, ON, Canada.
Department of Pediatrics, Faculty of Medicine, University of Toronto, Toronto, ON, Canada.


Pediatric-onset multiple sclerosis (MS) comprises 2-5% of MS cases, and is known to be associated with high disease activity and the accumulation of disability at an earlier age than their adult-onset counterparts. Appropriate therapy leading to disease control has the potential to alter the known trajectory of adverse long-term physical, cognitive, and psychosocial outcomes in this population. Thus, optimizing treatment for children and adolescents with MS is of paramount importance. The last decade has seen a growing number of disease-modifying therapies approved for relapsing MS in adults, and available agents now include oral, injectable, and infusion therapies. Recently, the development of randomized controlled MS trials in youth has led to the first agent approved by the US FDA for the treatment of pediatric MS-fingolimod. With this, we have entered a new era of knowledge and treatment in this population and ongoing pediatric trials are expected to further inform clinical management. With the emergence of highly effective therapies targeting the inflammatory component of the disease, there has been increased interest in identifying treatment strategies that instead target mechanisms such as remyelination/repair, neuroprotection, or rehabilitation. The potential role for such emerging therapies in the treatment of pediatric MS remains an important area of study. In this review, we discuss current evidence for MS therapies in children including the treatment of acute relapses, disease-modifying therapies, and symptomatic management. We will also discuss evidence for emerging therapies, including remyelinating and neuroprotective agents.


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