4.6 Review

Bringing the HEET: The Argument for High-Efficacy Early Treatment for Pediatric-Onset Multiple Sclerosis

Journal

NEUROTHERAPEUTICS
Volume 14, Issue 4, Pages 985-998

Publisher

SPRINGER
DOI: 10.1007/s13311-017-0568-1

Keywords

Pediatric-onset multiple sclerosis; disease-modifying therapy; neurodevelopment; natalizumab; rituximab; NEDA

Funding

  1. National Multiple Sclerosis Society [FP-1506-04742]

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Pediatric-onset multiple sclerosis (POMS) is rarer than adult-onset disease, and represents a different diagnostic and treatment challenge to clinicians. We review POMS clinical and radiographic presentations, and explore important differences between POMS and adult-onset MS natural histories and long-term outcomes. Despite having more active disease, current treatment guidelines for patients with POMS endorse the off-label use of lower-efficacy disease-modifying therapies (DMTs) as first line. We review the available MS DMTs, their evidence for use in POMS, and the contrasting treatment strategies of high-efficacy early treatment and escalation therapy. We introduce a new treatment approach, the high-efficacy early treatment, or HEET strategy, based on using directly observed, high-efficacy intravenously infused DMTs as first-line therapies. Like other proposed POMS treatment strategies, HEET will need to be prospectively studied, and all treatment decisions should be determined by an experienced neurologist, the patient, and his/her parents.

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