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Oral Dimethyl Fumarate in Children With Multiple Sclerosis: A Dual-Center Study

Naila Makhani1, Teri Schreiner2

  • 1Department of Pediatrics, Yale University School of Medicine, New Haven, Connecticut; Department of Neurology, Yale University School of Medicine, New Haven, Connecticut.

Pediatric Neurology
|March 22, 2016
PubMed

Insights

Oral dimethyl fumarate is a potentially safe and effective treatment for pediatric multiple sclerosis, with most children tolerating the medication and showing improved outcomes. Further clinical trials are underway to confirm efficacy.

Area of Science:

  • Neurology
  • Pediatrics
  • Immunology

Background:

  • Injectable therapies for pediatric multiple sclerosis (MS) may have limitations in efficacy and tolerability.
  • There is a critical need for effective oral treatment options for children with MS.
  • This study examines the use of oral dimethyl fumarate in pediatric MS patients.

Purpose of the Study:

  • To evaluate the safety and tolerability of oral dimethyl fumarate in children with multiple sclerosis.
  • To assess the preliminary efficacy of oral dimethyl fumarate in a pediatric MS cohort.
  • To review clinical, demographic, and MRI outcomes in children treated with dimethyl fumarate.

Main Methods:

  • Retrospective review of 13 pediatric patients (≤18 years) with MS treated with oral dimethyl fumarate.
  • Analysis of clinical data, demographic information, and serial magnetic resonance imaging (MRI).
  • Assessment of treatment tolerability, dose escalation, side effects, and relapse rates.

Main Results:

  • Dimethyl fumarate was used as first-line therapy in 38% of patients.
  • 89% of patients with ≥12 months follow-up showed stabilized or reduced relapse rates and disability.
  • Common side effects included flushing (62%) and gastrointestinal discomfort (54%); 23% discontinued treatment due to side effects. No serious adverse events like infections or laboratory abnormalities were reported.

Conclusions:

  • Oral dimethyl fumarate demonstrates a favorable safety and tolerability profile in children with multiple sclerosis.
  • Preliminary efficacy suggests stabilization or reduction in disease activity.
  • Ongoing formal clinical trials are necessary to definitively establish efficacy.
Abstract

Keywords:
Expanded Disability Status Scaledimethyl fumaratemultiple sclerosisoral therapy

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