Serum neurofilament light chain database effective in estimating pediatric neuro issues

Key takeaways:

• The study involved analysis of 2,667 healthy young persons and 220 young persons with a range of neurological conditions.
• Serum neurofilament light chain Z scores higher for neuro cohort, with higher effect size.

Compared with absolute values, serum neurofilament light chain Z scores were linked to higher effect size metrics and more accurate estimation of persistent neuroaxonal damage in pediatric populations, per data from The Lancet Neurology.

Lead study author Ahmed Abdelhak, MD, of the Weill Institute for Neurosciences at the University of California, San Francisco, and colleagues sought to create an age-adjusted serum neurofilament light (sNfL) reference range database for healthy children and adolescents, as well as to find validation for it by examining two additional cohorts of pediatric patients with a range of neurological conditions.

“The database is an invaluable tool for clinical trials exploring treatment methods related to neurological conditions,” Masoud Toloue, CEO of Quanterix, told Healio in an email. “Their research, supported by Quanterix’s Simoa technology, is paving the way for the expanded use of serum neurofilament (sNfL) in this age demographic and is providing pediatric clinicians with additional tools for capturing neuroaxonal injury.”

The study involved acquiring sNfL values from 2,667 healthy children and adolescents (median age 8 years; 50.1% girls) within two large European cohorts (Coronavirus Antibodies in Kids from Bavaria study, Germany) and North America (U.S. Network of Pediatric Multiple Sclerosis Centers pediatric case-control cohort). Abdelhak and colleagues employed linear models to retrospectively study the age and weight as determinants on sNfL concentrations, and modeled distribution of sNfL concentrations as a function of age-related physiological changes to derive reference percentile and Z score values.

Utility of the new reference dataset was then assessed in 220 young persons (median age 14.7 years; 60.9% girls) with a range of neurological diseases, taken from the pediatric neuroimmunology clinic at UCSF and the Children’s Hospital of the University of Regensburg in Germany.

Results showed that sNfL concentrations decreased with age by an estimated 6.8% per year until age 10.3 years (estimated multiplicative effect per 1 year increase 0.93 [95% CI, 0.93–0.94]) in the healthy population and was mostly stable up to age 22 (1.00 [0.52–1.94]). Independent of age, researchers found a marginal effect of weight on sNfL concentrations.

Data further showed, in the 220 youths with neurological conditions, age-adjusted sNfL Z scores were higher than the reference population of healthy subjects with higher effect size metrics (Cohen’s d = 1.56) compared with the application of raw sNfL concentrations (d = 1.28).

“Clinicians are now empowered to utilize blood NfL measurements as an aid in assessing neurological disease activity in children,” Toloue told Healio. “The clinical potential and impact of blood NfL continues to expand, both as a single assay and in combination with other biomarkers.”