Highly Effective Therapies Found Superior to Moderately Effective Therapies as First-Line Treatment in Pediatric MS

Onset and diagnosis of multiple sclerosis (MS) typically occur between the ages of 20 and 40 years. When the disease is diagnosed before the age of 18, it is considered pediatric-onset MS (POMS), and in 3 to 5% of people with MS, onset occurs before 16 years of age.

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People with POMS have two to three times more frequent relapses during the first six years after diagnosis than adults with early-onset MS. They are also more likely to have significant disability at an age ten years younger than adults with early-onset MS.

As with adult-onset MS, early treatment of POMS is essential to halt disease progression, including early disability, brain volume loss, and cognitive impairment. Currently, the only disease-modifying therapies FDA-approved for use in children with MS are Gilenya and Tascenso ODT, both brand-name versions of the generic fingolimod. Other treatments prescribed for children with MS are used off-label.

It is common practice with both adults and children with MS to begin treatment with moderately effective therapies and move up to highly effective therapies if the patient does not respond to the treatment or it stops working. Examples of moderately effective therapies include azathioprine, glatiramer acetate, dimethyl fumarate, interferon beta, and teriflunomide. Highly effective therapies include fingolimod, Lemtrada (alemtuzumab), Tysabri (natalizumab), Ocrevus (ocrelizumab), and Kesimpta (ofatumumab).

Little is known about the outcome of different treatment strategies in patients with POMS. In a study published recently in JAMA Neurology, Nail Benallegue, M.D., Ph.D., from the department of pediatric neurology at the University of Angers in France, and his colleagues used records from the national French MS cohort Observatoire Français de la Sclérose en Plaques (OFSEP) to analyze the real-world outcomes of using highly effective therapies as an index treatment versus using moderately effective therapies in patients with POMS.

The study included patients with POMS who received initial treatment before the age of 18 years between January 1, 2010, and December 8, 2022. Of 530 patients included, 422 initiated treatment with a moderately effective therapy, and 108 began with a highly effective therapy. The most used moderately effective therapies were interferon beta, glatiramer acetate, and dimethyl fumarate. Fingolimod and Tysabri were the most commonly used highly effective therapies.

The study’s primary outcome was the time to first relapse after treatment. Secondary outcomes included annual relapse rate, magnetic resonance imaging activity, time to Expanded Disability Status Scale (EDSS) progression, higher education attainment, and incidence of serious adverse events.

After a median follow-up of 5.8 years, the researchers found that both treatment strategies significantly reduced relapses during the first two years. However, children who started treatment with a highly effective therapy had a 54% lower risk of first relapse at five years compared with those who began treatment with a moderately effective therapy.

Benallegue and his colleagues concluded, “The findings of this cohort study suggest a sustained reduction in disease activity over 5 years associated with use of [a highly effective therapy] as the primary strategy in POMS, with an optimal impact within the first 2 years.”

They emphasized the need for long-term safety studies and added, “The present findings corroborate current expert opinions and suggest prioritizing initial [highly effective therapy] in children with POMS.”