Natalizumab for pediatric multiple sclerosis: a systematic review and meta-analysis

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.