Based on findings from the study, investigators stressed the need for better streamlined access to high-efficacy therapies for pediatric patients with onset multiple sclerosis.
Sifat Sharmin, PhD
(Credit: University of Melbourne)
A new study showed that starting monoclonal antibody therapy during childhood was associated with reduced disability at age 23 and beyond, compared with initiating treatment later in the course of the disease, among pediatric patients with multiple sclerosis (MS).1,2 These findings suggest that earlier treatment with these highly effective therapies, prior to the onset of significant neurological impairments, is essential for preserving long-term neurological function in pediatric patients with relapsing-remitting MS.
Among 282 patients with pediatric-onset MS, 110 started therapy early (39%) and 172 commenced treatment late (61%). Presented at the 2024 European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress, September 18-20, in Copenhagen, Denmark, researchers reported that disability was 0.57 steps lower on Expanded Disability Status Scale (EDSS) in the patient group that received treatment earlier compared with the group that initiated treatment later, between the ages of 23 to 27 years (β -0.57; 95% CI, -0.90 to -0.23).
“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%,” lead author Sifat Sharmin, PhD, research fellow in the Clinical Outcomes Research Unit at the University of Melbourne, said in a statement.2 “This study highlights the critical importance of early intervention in pediatric-onset MS. 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.”
Investigators identified patients who experienced the onset of MS symptoms under the age of 18 years from the French MS Registry, Italian MS Register, and the global MSBase Registry. Researchers included patients who had a follow-up of at least age 23 and who started monoclonal antibody treatment such as ocrelizumab (Ocrevus; Roche), rituximab (Rituxan; Genentech), or natalizumab (Tysabri; Biogen) either between the ages of 12 to 17 years or 20 to 22 years.
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Authors had inverse probability treatment weights estimated based on patients’ baseline clinical and demographic characteristics at the age of 18 years. The primary outcome evaluated was the change in EDSS scores from baseline to ages 23 to 27 years, which was assessed using a Bayesian weighted generalized linear mixed-effect model.
Additional findings revealed that the mean absolute differences in EDSS from baseline were 0.40 in the early treatment group and 0.95 in the late treatment group. Authors noted that this benefit of early treatment was consistent during the rest of the follow-up period. At the baseline age of 18 years, authors reported that the median EDSS score was 1.5 (1st-3rd quartiles 1.0-2.5) in the early treatment group and 1.3 (quartiles 0.0-2.0) in the late treatment group. All told, the median follow-up time was 10.8 years (quartiles 8.5-14.0).
Regulatory restrictions currently often delay access to these high effective treatments for pediatric patients with onset MS until adulthood because of limited evidence of the efficacy, safety, and impact of monoclonal antibodies on their development.3 “These findings are a strong argument for rethinking current treatment guidelines,” Sharmin said in a statement.2 “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.” Researchers noted they are committed to producing additional evidence to support proactive treatment of pediatric-onset MS, focusing specifically on assessing long-term risks of immunosuppressive therapies in this patient population.
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