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Newer Treatment Approaches in Pediatric-Onset Multiple Sclerosis

  • Gabrielle Macaron
  • Jenny Feng
  • Manikum Moodley
  • Mary RenselEmail author
Multiple Sclerosis and Related Disorders (J Graves, Section Editor)
Part of the following topical collections:
  1. Topical Collection on Multiple Sclerosis and Related Disorders

Abstract

Purpose of review

With the recognition that pediatric-onset multiple sclerosis (POMS) is characterized by more prominent disease activity, earlier age at onset of disability milestones, and more prominent cognitive impairment compared with physical disability earlier in the disease course compared with adult-onset multiple sclerosis (AOMS), there has been increasing interest in identifying optimal and safe treatment approaches to achieve better disease control in this group. Injectable therapies have been traditionally used as first line in this population, although not formally approved. This review focuses on current treatment and monitoring approaches in POMS.

Recent findings

In the past few years, and despite the paucity of FDA-approved medications for use in POMS, an increasing trend toward using newer disease-modifying therapies (DMTs) in this group is observed. However, escalation (as opposed to induction) remains the most frequent approach, and many children continue to be untreated before age 18, particularly before age 12. The only FDA- and EMA-approved disease-modifying therapy in POMS is fingolimod; however, dimethyl fumarate, teriflunomide, natalizumab, ocrelizumab, and alemtuzumab either have been evaluated in observational studies or are being currently investigated in formal randomized controlled trials for use in POMS and appear to be safe in this group. Autologous hematopoietic stem cell transplantation has also been evaluated in a small series. Clinical outcome measures and MS biomarkers have been poorly studied in POMS; however, the use of composite functional scores, neurofilament light chain, optical coherence tomography, and imaging findings is being increasingly investigated to improve early diagnosis and efficient monitoring of POMS.

Summary

Off-label use of newer DMTs in POMS is increasing, and based on retrospective data, and phase 2 trials, this approach appears to be safe in children. Results from ongoing trials will help clarify the safety and efficacy of these therapies in the future. Fingolimod is the only FDA-approved medication for use in POMS. Outcome measures and biomarkers used in AOMS are being studied in POMS and are greatly needed to quantify treatment response in this group.

Keywords

Multiple sclerosis Pediatric Treatment Outcome measures Safety Efficacy 

Notes

Compliance with Ethical Standards

Conflict of Interest

Gabrielle Macaron receives fellowship funding from the National Multiple Sclerosis Society Institutional Clinician Training Award ICT 0002 and has received fellowship funding from Biogen Fellowship Grant 6873-P-FEL. She has served on a scientific advisory board for Genentech.

Jenny Feng receives funding via a Sylvia Lawry Physician Fellowship Grant through the National Multiple Sclerosis Society (No. FP-1707-28768). She has served on scientific advisory board for Sanofi.

Manikum Moodley receives funding from the National Multiple Sclerosis Society.

Mary Rensel serves as a consultant or speaker for Biogen, Teva, Genzyme, and Novartis. She receives grant funding from the National Multiple Sclerosis Society. She also serves on the advisory board for Serono and received research support from MedImmune and Genentech.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.

References and Recommended Reading

Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2019

Authors and Affiliations

  • Gabrielle Macaron
    • 1
  • Jenny Feng
    • 1
  • Manikum Moodley
    • 2
  • Mary Rensel
    • 1
    Email author
  1. 1.Mellen Center for Multiple Sclerosis, Cleveland ClinicClevelandUSA
  2. 2.Center for Pediatric Neurosciences, Cleveland ClinicClevelandUSA

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