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Rituximab treatment in pediatric-onset multiple sclerosis.

Markus Breu1, Fredrik Sandesjö2, Ruxandra-Iulia Milos3

  • 1Division of Pediatric Pulmonology, Allergology and Endocrinology, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria.

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Summary

Rituximab (RTX) significantly reduced relapses and MRI lesions in pediatric multiple sclerosis patients. Most patients achieved no evidence of disease activity, though adverse events were common.

Keywords:
central nervous systemcohort studydisease‐modifying therapypediatric‐onset multiple sclerosisrituximab

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Area of Science:

  • Neurology
  • Immunology
  • Pediatric Medicine

Background:

  • Rituximab (RTX) is widely used for multiple sclerosis (MS).
  • Limited data exists on RTX efficacy and safety in pediatric-onset MS.
  • Understanding RTX's risk-benefit profile in young MS patients is crucial.

Purpose of the Study:

  • To evaluate the efficacy and safety of Rituximab (RTX) in pediatric-onset multiple sclerosis.
  • To assess RTX's impact on clinical relapse rates and MRI activity.
  • To determine the proportion of patients achieving no evidence of disease activity (NEDA-3).

Main Methods:

  • Retrospective cohort study of 61 pediatric MS patients from Sweden, Austria, and Germany treated with RTX.
  • Assessed annualized relapse rates, Expanded Disability Status Scale (EDSS), and MRI parameters (T2 lesions, contrast-enhancing lesions) pre- and post-treatment.
  • Monitored adverse events, including infusion reactions and infections.

Main Results:

  • RTX treatment led to a significant reduction in annualized relapse rates (0.6 to 0.03).
  • MRI activity substantially decreased, with annual new T2 lesions dropping from 1.25 to 0.08 and contrast-enhancing lesions from 0.86 to 0.
  • 70% of patients achieved NEDA-3; 67% experienced adverse events, with 6 discontinuing treatment.

Conclusions:

  • Rituximab (RTX) demonstrates significant efficacy in reducing clinical and radiological disease activity in pediatric-onset multiple sclerosis.
  • RTX is a viable treatment option for pediatric MS, offering substantial disease control.
  • Careful monitoring for adverse events is recommended during RTX therapy in this population.