DOI: 10.1177/13524585261450816 ISSN: 1352-4585

Serum neurofilament light chain in paediatric patients treated with natalizumab for highly active multiple sclerosis

Brenda Huppke, Marie-Christine Reinert, Wiebke Stark, Jutta Gärtner, Peter Huppke

Background:

Natalizumab (NTZ) effectively suppresses clinical and radiologic disease activity in paediatric patients with multiple sclerosis (MS), however, it is unclear whether serum neurofilament light chain (sNfL), a biomarker of neuronal injury, normalizes.

Objective:

To evaluate sNfL response to NTZ in the paediatric setting.

Methods:

sNfL was measured (single-molecule array) in 66 paediatric patients on NTZ for highly active MS in a single-centre between 2007 and 2023. sNfL levels were converted to age-adjusted z -scores for longitudinal assessment.

Results:

sNfL declined significantly on NTZ, however, not all patients achieved normalized levels. Percentage of patients with sNfL < 90th percentile of healthy aged-matched individuals increased from 6.8% initially to 50%, 65% and 75% within 12, 24 and 36 treatment-months, respectively. Higher on-treatment levels were associated with higher baseline levels ( B  =  0 .791, p  < 0.001) and John Cunningham virus (JCV) seropositivity ( B  = 0.286, p  < 0.001). sNfL > 97th percentile at 12 treatment-months indicated greater likelihood of disease activity in following year.

Conclusions:

NTZ treatment resulted in a significant reduction in sNfL levels, correlating with its clinical efficacy in paediatric MS. However, higher baseline sNfL concentrations were associated with a prolonged time to normalization. In addition, JCV seropositivity was linked to elevated NfL levels during treatment.

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