Almost all men with ALD develop a slowly progressive spinal cord disease called myelopathy, but the rate at which this occurs varies considerably from person to person. Identifying those who will experience a faster progression of symptoms is essential for clinical care and the design of clinical trials. This study investigated whether two measurable blood proteins — neurofilament light chain (NfL) and glial fibrillary acidic protein (GFAP) — could serve as prognostic biomarkers. In a seven-year prospective longitudinal study, 66 adult men with ALD and no cerebral involvement were observed.[Click to read more ▼]
Plasma NfL and GFAP levels were measured using highly sensitive Simoa technology, and patients were stratified based on their baseline levels. Men with high baseline NfL levels showed significantly faster worsening on disability and spinal cord outcome measures, with differences already apparent within the first year of follow-up. Importantly, plasma NfL levels remained stable over time, indicating that a single measurement has lasting predictive value. GFAP stratification, by contrast, showed limited prognostic utility on its own. Baseline plasma NfL is a robust and practical biomarker for identifying men with ALD who are at higher risk for faster spinal cord disease progression. This supports the use of NfL for patient stratification in future clinical trials, making those trials more efficient and better able to detect treatment effects.