CSF neurofilament light chain is elevated in OMS (decreasing with immunotherapy) and other pediatric neuroinflammatory disorders

Abstract Using a panel of seven brain cell-specific biomarkers in cerebrospinal fluid (CSF), pediatric opsoclonus–myoclonus syndrome (OMS) ( n = 234) was compared to pediatric non-inflammatory neurological controls ( n = 84) and other inflammatory neurological disorders (OIND) ( n = 44). Only CSF NFL was elevated in untreated OMS versus controls (+ 83%). It was 87% higher in OIND than in OMS. On combination treatment with front-loaded ACTH, IVIg, rituximab, median CSF NFL decreased by 60% to control levels. These biochemical data suggest neuronal/axonal injury in some children with OMS without indicators of astrogliosis, and reduction on sufficient immunotherapy.