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Efficacy and safety of ocrelizumab in pediatric multiple sclerosis
Ahmad Altelly1, Azza Mahmoud2, Victoria Mifsud2
1College of Medicine, University of Sharjah, Sharjah, United Arab Emirates.
Insights
Ocrelizumab shows promise for pediatric multiple sclerosis (MS) by significantly reducing relapses and maintaining disability. Further research is needed to confirm long-term safety and effectiveness in young patients.
Area of Science:
- Neurology
- Immunology
- Pediatric Medicine
Background:
- Limited data exists on ocrelizumab, an anti-CD20 monoclonal antibody, for pediatric-onset multiple sclerosis (POMS).
- This study addresses the safety and efficacy of ocrelizumab in POMS patients within the UAE.
Purpose of the Study:
- To evaluate the safety and efficacy of ocrelizumab in pediatric patients diagnosed with multiple sclerosis.
- To assess relapse rates, disability progression (EDSS), MRI findings, and adverse events in POMS patients treated with ocrelizumab.
Main Methods:
- Retrospective cohort study analyzing electronic medical records of 29 POMS patients treated with ocrelizumab before age 18.
- Data included demographics, disease history, prior treatments, relapse rates, MRI, IgG levels, and EDSS scores.
Main Results:
- Ocrelizumab treatment led to a 97% reduction in annualized relapse rate (ARR) in relapsing-remitting MS patients.
- Expanded Disability Status Scale (EDSS) scores remained stable; mild infusion reactions and infections were reported.
- Immunoglobulin G (IgG) levels were stable, with no new MRI lesions or relapses during treatment.
Conclusions:
- Ocrelizumab demonstrated good short-term tolerability and efficacy in POMS patients, significantly reducing relapses.
- Stable disability and manageable adverse events suggest potential benefit, but larger prospective studies are required for long-term validation.
Introduction:
Ocrelizumab, a highly effective anti-CD20 monoclonal antibody, is approved for adult use, but pediatric data remain limited. This retrospective cohort study evaluates its safety and efficacy in pediatric-onset multiple sclerosis (POMS) patients at two tertiary hospitals in the UAE-Cleveland Clinic Abu Dhabi (CCAD) and Sheikh Shakhbout Medical City (SSMC).
Methods:
Electronic medical records of patients treated with ocrelizumab before age 18 were reviewed. Data collected included demographics, disease characteristics, use of prior disease-modifying therapies, relapse rates, MRI findings, immunoglobulin G (IgG) levels, and Expanded Disability Status Scale (EDSS) scores.
Results:
Twenty-nine POMS patients (19 CCAD, 10 SSMC) received ocrelizumab before age 18. The cohort was predominantly female (6.25:1 ratio) and UAE nationals (83%). Most had relapsing-remitting MS (97%), with a mean onset age of 14.6 years and a mean treatment initiation age of 15.6 years. Median time from ocrelizumab initiation to last follow-up was 15 months. The annualized relapse rate (ARR) in the RRMS group decreased by 97% post-treatment. EDSS scores remained stable. Two patients experienced mild infusion reactions. Nine (31%) had infections post-treatment, including four COVID-19 cases. One relapse occurred while off treatment; two patients had new MRI lesions. IgG levels remained stable.
Conclusion:
In this cohort, ocrelizumab was well-tolerated with reduced relapse rates and stable disability scores over the short-term follow-up. Larger, prospective studies with longer-term data are needed to confirm safety and efficacy.
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