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Dynamic Imaging of Chimeric Antigen Receptor T Cells with [18F]Tetrafluoroborate Positron Emission Tomography/Computed Tomography
Published on: February 17, 2022
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.
Insights
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.
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.
Background And Purpose:
Rituximab (RTX) is frequently used off-label in multiple sclerosis. However, studies on the risk-benefit profile of RTX in pediatric-onset multiple sclerosis are scarce.
Methods:
In this multicenter retrospective cohort study, patients with pediatric-onset multiple sclerosis from Sweden, Austria and Germany, who received RTX treatment were identified by chart review. Annualized relapse rates, Expanded Disability Status Scale scores and magnetic resonance imaging parameters (new T2 lesions and contrast-enhancing lesions) were assessed before and during RTX treatment. The proportion of patients who remained free from clinical and disease activity (NEDA-3) during RTX treatment was calculated. Side effects such as infusion-related reactions, infections and laboratory abnormalities were assessed.
Results:
Sixty-one patients received RTX during a median (interquartile range) follow-up period of 20.9 (35.6) months. The annualized relapse rate decreased from 0.6 (95% confidence interval [CI] 0.38-0.92) to 0.03 (95% CI 0.02-0.14). The annual rate of new T2 lesions decreased from 1.25 (95% CI 0.70-2.48) to 0.08 (95% CI 0.03-0.25) and annual rates of new contrast-enhancing lesions decreased from 0.86 (95% CI 0.30-3.96) to 0. Overall, 70% of patients displayed no evidence of disease activity (NEDA-3). Adverse events were observed in 67% of patients. Six patients discontinued treatment due to ongoing disease activity or adverse events.
Conclusion:
Our study provides class IV evidence that RTX reduces clinical and radiological activity in pediatric-onset multiple sclerosis.
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