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Natalizumab for pediatric multiple sclerosis: a systematic review and meta-analysis
Yasin Ebne-Ali-Heydari1, Aryana Ramezani2, Amirmohammad Jozayi1
1School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran.
Insights
Natalizumab significantly reduces relapses and disability in pediatric-onset multiple sclerosis (POMS). This higher-efficacy treatment (HET) shows an acceptable safety profile, making it a viable option for POMS patients.
Area of Science:
- Neurology
- Immunology
- Pharmacology
Background:
- Pediatric-onset multiple sclerosis (POMS) affects individuals under 18, representing 3-5% of all MS cases.
- Natalizumab (NTZ) is a high-efficacy disease-modifying treatment increasingly utilized for POMS.
Purpose of the Study:
- To systematically review and meta-analyze the efficacy and safety of natalizumab in POMS.
- To provide quantitative data on relapse rates, disability progression, adverse events, and JC virus seropositivity.
Main Methods:
- A comprehensive literature search was conducted across PubMed, Embase, Web of Science, and Scopus (1991-2025).
- Included 18 non-randomized interventional studies with 922 POMS patients.
- Primary endpoint: mean difference in annualized relapse rate (ARR); Secondary outcomes: Expanded Disability Status Scale (EDSS) changes, adverse events (AEs), and JC virus seropositivity.
Main Results:
- Natalizumab therapy demonstrated a significant mean reduction in ARR of -1.962 relapses/patient-year (p<0.001).
- A statistically significant improvement in disability was observed, with a mean EDSS change of -0.807 (p<0.001).
- AEs occurred in 18% of patients, and JC virus seropositivity was noted in 12%; no progressive multifocal leukoencephalopathy (PML) cases were reported.
Conclusions:
- Natalizumab is a potentially effective therapeutic option for POMS patients with highly active disease or inadequate response to other treatments.
- The safety profile of natalizumab in POMS is considered acceptable, with manageable adverse events.
Background:
Pediatric-onset multiple sclerosis (POMS) is the onset of MS before the age of 18 and accounts for 3%-5% of all multiple sclerosis (MS) cases. Natalizumab (NTZ) is among the higher-efficacy disease-modifying treatments (HETs) in MS and is increasingly used for POMS.
Objectives:
In this systematic review and meta-analysis, we aimed to discuss the debate on the efficacy and safety of natalizumab use in POMS, providing quantitative results on relapse rate, disability progression, adverse events (AEs), and JC virus seropositivity.
Design:
The primary endpoint for meta-analysis was the mean difference (MD) in annualized relapse rate (ARR) after natalizumab compared to before treatment. Secondary outcomes were the MD of Expanded Disability Status Scale (EDSS) and the proportion of POMS patients experiencing AEs and JC virus seropositivity after natalizumab treatment.
Data Sources And Methods:
We performed a comprehensive search of PubMed, Embase, Web of Science, and Scopus between January 1, 1991 and May 1, 2025.
Results:
In this systematic review, 18 non-randomized interventional studies including 922 patients with POMS were included. Natalizumab therapy was associated with a mean reduction in ARR of -1.962 relapses per patient-year from baseline (95% confidence interval (CI): -2.449 to -1.475; p < 0.001). The drug was also associated with a statistically significant improvement in disability, with a mean change in EDSS of -0.807 from baseline (95% CI: -1.078 to -0.536; p < 0.001). After treatment, 18% of patients experienced AEs (95% CI: 0.11-0.25), and JC virus seropositivity was observed in 12% (95% CI: 0.07-0.17). No case of progressive multifocal leukoencephalopathy was reported among the 922 natalizumab-treated patients.
Conclusion:
Natalizumab may represent a viable therapeutic option for POMS patients exhibiting highly active disease or serves as an effective alternative in those with inadequate response to initial treatment. The safety profile remains acceptable, with most AEs being manageable.
Registration:
PROSPERO (CRD42024583911). This study follows the Preferred Reporting Items for Systematic Reviews and Meta-Analyses reporting guidelines.
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