Real-World Study Analyzes Which First-Line Therapy is Most Effective in Pediatric-Onset Multiple Sclerosis

According to results of a study published in JAMA Neurology, initiating highly effective therapies (HETs) as a first-line treatment for pediatric-onset multiple sclerosis (POMS) may be beneficial as compared with moderately effective therapies (METs). The use of HETs was shown to be associated with significant reductions in risk of first relapse and demonstrated better tolerability over a 5-year period compared with METs. The findings suggest that the primary strategy for treating people with POMS, which currently constitutes the initiation of METs, might be improved through the potential implementation of HETs as a first-line treatment.

The findings result from a retrospective, observational cohort study conducted from 2010 to 2022 based on data from 36 expert centers in the French Multiple Sclerosis (MS) database, the Observatoire Française de la Sclérose en Plaques (OFSEP). The study included 530 treatment-naïve children with relapsing-remitting POMS who received a first treatment before they were aged 18 years. Participants were categorized as starting treatment with HETs or METs.

In this study, HETs included:

• Lemtrada (alemtuzumab; Sanofi, Bridgewater, NJ)
• Gilenya (fingolimod; Novartis, East Hanover, NJ)
• Mavenclad (cladribine; Merck, Darmstadt, Germany)
• Tysabri (natalizumab; Biogen, Cambridge, MA)
• Ocrevus (ocrelizumab; Genentech, South San Francisco, CA)
• Kesimpta (ofatumumab; Novartis, East Hanover, NJ)
• Rituximab
• Mitoxantrone

The primary outcome measure was time to first relapse, and the average follow up was 5.8 years. After adjusting for treatment initiation epoch, participants who started on HETs were shown to have a 54% lower risk of first relapse compared to those who started on METs (adjusted hazard ratio [HR], 0.46; 95% CI, 0.31 to 0.67; P<.001), which was sustained over 5 years. Participants in the HET group also showed improvements in the secondary endpoint of MRI activity (adjusted odds ratio [OR], 0.34; 95% CI, 0.18 to 0.66; P=.001). Additionally, risk of discontinuation at 2 years was 6 times higher for participants who started on METs as compared with HETs (HR, 5.97; 95% CI, 2.92 to 12.20).