Early intervention in pediatric-onset MS can lead to improved long-term outcomes

New research presented today at ECTRIMS 2024 reveals that initiating monoclonal antibody therapy during childhood, rather than delaying treatment until early adulthood, significantly reduces long-term disability in multiple sclerosis (MS) patients.

The study, which utilized data from the French MS Registry, Italian MS Register, and the global MSBase Registry, analyzed the outcomes of 282 patients with pediatric-onset MS who began experiencing symptoms before the age of 18 years. Patients were divided into two groups based on when they initiated monoclonal antibody treatment: either between the ages of 12-17 or 20-22 years.

To ensure comparability between the groups, the researchers used inverse probability treatment weighting based on propensity scores, which accounted for baseline differences in factors such as sex, age at symptom onset, time from onset to clinically definite MS, and the number of relapses. This approach enabled a clear assessment of how the timing of initiating high-efficacy therapy affects disability outcomes from ages 23 and over.

Using the Expanded Disability Status Scale (EDSS) to measure and monitor disability progression in MS, the study showed that patients who began treatment between the ages of 12-17 years (39% of the study group) had a mean absolute increase of only 0.40 points on the EDSS, compared to a 0.95-point increase in those who started treatment later (61% of the study group).

Between the ages of 23 and 27, the increase in EDSS scores from baseline was 0.57 points lower in the early treatment group compared to the late treatment group. The benefits of early treatment persisted throughout the median follow-up period of 10.8 years.

The substantially lower risk of progressing to higher disability levels in the early treatment group was particularly evident in the moderate disability range, where further progression was reduced by up to 97%."

Dr. Sifat Sharmin, research fellow in the Clinical Outcomes Research (CORe) Unit, University of Melbourne and leader of the study

"This study highlights the critical importance of early intervention in pediatric-onset MS," emphasizes Dr. Sharmin. "Our findings indicate that initiating high-efficacy therapies like ocrelizumab, rituximab, or natalizumab during childhood can lead to significantly improved long-term outcomes, preserving neurological function and reducing disability progression."

Currently, regulatory restrictions, due to limited evidence of the efficacy, safety, and impact of monoclonal antibodies on children's development, often delay access to these treatments for pediatric-onset MS patients until adulthood. "These findings are a strong argument for rethinking current treatment guidelines," Dr. Sharmin urges. "By allowing earlier access to effective treatments, we can significantly enhance the quality of life for children with MS and reduce the burden of long-term disability."

Looking ahead, the research team are dedicated to generating further evidence to support the proactive treatment of pediatric-onset MS, with a particular focus on assessing the long-term risks of immunosuppressive therapies in this population.