Teriflunomide: an oral therapy for first-line treatment of children and adolescents living with relapsing-remitting multiple sclerosis

ABSTRACT

Introduction

Different disease-modifying therapies (DMTs) have been developed to slow down the progression of pediatric multiple sclerosis (MS). Teriflunomide is one such DMT that has recently been approved for use in pediatric MS in the European Union.

Areas covered

The article provides an introduction to the mechanism of action of teriflunomide, reviews the clinical trials conducted on the safety and efficacy of the drug, and the optimal dosing and monitoring strategies.

Expert opinion

Teriflunomide is an oral medication that has shown promise in improving outcomes for pediatric MS patients, including reduced relapse rates and improved quality of life. However, more research is needed to determine its long-term safety in pediatric patients. As MS often presents with an aggressive course in children, the choice of disease-modifying treatment should be carefully evaluated, with a preference for second-line therapy. Despite the potential benefits of teriflunomide, changes in clinical practice may be hindered by factors such as cost and physician familiarity with alternative treatments. Longer-term studies and biomarker identification are areas for improvement, but the future of research in this area holds promise for the continued development and refinement of disease-modifying therapies and more personalized, targeted treatments for pediatric MS patients.

KEYWORDS:

• Pediatric MS
• Teriflunomide
• Relapsing-remitting course
• Efficacy
• Safety
• Demyelination

Article highlights

• Pediatric multiple sclerosis (POMS) is a rare autoimmune disease affecting children and adolescents that manifests before the age of 16.

• POMS, compared to adult MS, is characterized by a more aggressive disease onset, higher relapse rate, and a polyfocal presentation at disease onset.

• Early intervention with disease-modifying therapies (DMTs) is important to reduce the risk of permanent disability.

• Teriflunomide is an oral immunomodulatory drug that has shown efficacy in reducing annualized relapse rates in adult patients with relapsing-remitting MS and has been approved by the EMA for use in pediatric patients.

• The TERIKIDS trial showed that teriflunomide is effective in reducing magnetic resonance activity and biomarkers of axonal damage in children with relapsing MS.

• Teriflunomide is generally well tolerated and has a favorable safety profile in both adults and pediatric patients, but there are some safety concerns such as hepatotoxicity, pancreatitis, and teratogenicity that need to be monitored.

• Fingolimod has also been approved for treatment of pediatric MS, and the choice between teriflunomide and fingolimod should be based on the individual prognostic profile.

Declaration of interest

G Comi has received personal compensation outside of the submitted work from Novartis, Teva Pharmaceutical Industries Ltd, Teva Italia Srl, Sanofi Genzyme, Genzyme Corporation, Genzyme Europe, Merck KGgA, Merck Serono SpA, Celgene Group, Biogen Idec, Biogen Italia Srl, F. Hoffman-La Roche, Roche SpA, Almirall SpA, Forward Pharma, Medday, and Excemed. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

One referee has served on the clinical trial advisory board for the TERIKIDS clinical trial sponsored by Sanofi. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Additional information

Funding

This paper was not funded.