Consensus on the first-line treatment of pediatric-onset multiple sclerosis (POMS) remains unresolved. Recently, dimethyl fumarate (DMF) and anti-CD20 therapies have been among the favorable options for pediatric multiple sclerosis (MS).

This study aimed to determine the effectiveness and safety of DMF versus anti-CD20 therapies for POMS.

We conducted a retrospective cohort study from July 2012 to July 2022 in the Isfahan MS clinic. MS cases under the age of 18 years old who received DMF or anti-CD20 agents as first-line treatment and were followed for at least 12 months were included.

About 124 POMS cases were screened, of which 39 met the inclusion criteria. About 23 patients received DMF, while 16 patients received anti-CD20 (rituximab or ocrelizumab). The median (interquartile range, IQR) annualized relapse rate (ARR) decreased significantly (both with p < 0.0001) from 2.63 (0.68) to 0.0 (1.0) in the DMF group and from 2.89 (1.39) to 0.0 (1.0) in the anti-CD20 group. The median (IQR) expanded disability status score insignificantly changed from 1.0 (1.0) to 1.0 (0.5) in the DMF group, while it changed from 1.25 (1.0) to 1.0 (0.5) in the anti-CD20 group. After 12 months of follow-up, 12/16 in the anti-CD20 group and 17/23 in the DMF group were relapse-free. None of the treatment outcomes were different between the two treatment cohorts. Our study also assessed adverse events (AEs).

While subject to replication in future clinical trials, both DMF and anti-CD20 therapies had a significant effect on reducing ARR and disease activity with an acceptable safety profile, but in our study, there were no differences in their efficacy.

The datasets generated and/or analyzed during the current study are not publicly available to ensure the privacy protection of the participants, but are available from the corresponding author on reasonable request.

M.E. and M.S. were involved in the conceptualization of the study. H.K. and M.E. contributed to the design of the work. H.K., A.M.J., and M.G. contributed to the acquisition and analysis of the data. H.K., A.R., P.M., and N.S. contributed to the interpretation of data and drafting the initial manuscript. All of the authors contributed to the critical/intellectual review of the initial manuscript, and all have approved and accepted to be accountable for the integrity of the final manuscript.

Informed consent was obtained from all of the study participants or their parents or legal guardians, and all methods were carried out in accordance with relevant guidelines and regulations. The study protocol was registered and gained all the necessary approvals from the Isfahan University of Medical Sciences ethics committee (Approval ID: IR.MUI.MED.REC.1400.742).

Received: 25 April 2025

Accepted: 13 August 2025

Accepted Manuscript online:
14 August 2025

Article published online:
25 August 2025

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