Serum Glial Fibrillary Acidic Protein: a Neuromyelitis Optica Spectrum Disorder Biomarker

Orhan Aktas, Michael A Smith, William A Rees, Jeffrey L Bennett, Dewei She, Eliezer Katz, Bruce A C Cree
Annals of Neurology 2021 March 16

OBJECTIVE: Blood tests to monitor disease activity, attack severity, or treatment impact in neuromyelitis optica spectrum disorder (NMOSD) have not been developed. This study investigated the relationship between serum glial fibrillary acidic protein (sGFAP) concentration and NMOSD activity and assessed the impact of inebilizumab treatment.

METHODS: N-MOmentum was a prospective, multicenter, double-blind, placebo-controlled, randomized clinical trial, in adults with NMOSD. sGFAP levels were measured by single-molecule arrays (SIMOA) in 1260 serial and attack-related samples from 215 N-MOmentum participants (92% aquaporin 4-immunoglobulin G-seropositive) and in control samples (from healthy donors and patients with relapsing-remitting multiple sclerosis).

RESULTS: At baseline, 62 participants (29%) exhibited high sGFAP concentrations (≥170 pg/mL; ≥2 standard deviations above healthy donor mean concentration) and were more likely to experience an adjudicated attack than participants with lower baseline concentrations (hazard ratio [95% confidence interval], 3.09 [1.6-6.1], p = 0.001). Median (interquartile range [IQR]) concentrations increased within 1 week of an attack (baseline: 168.4 [128.9-449.7] pg/mL; attack: 2160.1 [302.7-9455.0] pg/mL; p = 0.0015) and correlated with attack severity (median fold change from baseline (FC) [IQR], minor attacks: 1.06 [0.9-7.4]; major attacks: 34.32 [8.7-107.5]; p = 0.023). This attack-related increase in sGFAP occurred primarily in placebo-treated participants (FC: 20.2 [4.4-98.3]; p = 0.001) and was not observed in inebilizumab-treated participants (FC: 1.1 [0.8-24.6]; p > 0.05). Five participants (28%) with elevated baseline sGFAP reported neurological symptoms leading to non-adjudicated attack assessments.

INTERPRETATION: sGFAP may serve as a biomarker of NMOSD activity, attack risk, and treatment effects. (NCT02200770). This article is protected by copyright. All rights reserved.

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