# The Clinical Characteristics and Treatment of Patients With Autoimmune Glial Fibrillary Acidic Protein Astrocytopathy (GFAP‐A): A Retrospective Study of 29 Patients

## TL;DR

This study analyzed 29 patients with autoimmune GFAP astrocytopathy, finding that immunotherapy was effective and most patients had good outcomes.

## Contribution

The study provides new clinical insights into treatment efficacy and outcomes for GFAP-A patients.

## Key findings

- Immunotherapy was effective for both short- and long-term prognosis in GFAP-A.
- Most patients (25/29) had favorable outcomes despite disease severity.
- Non-pulse and pulse steroid therapies showed comparable efficacy.

## Abstract

To investigate the clinical features, treatment, and outcome of patients with autoimmune glial fibrillary acidic protein astrocytopathy (GFAP‐A).

Medical records and collected case data from the First Affiliated Hospital of Zhejiang University School of Medicine from November 2020 to May 2024 and retrospectively analyzed the clinical features, radiological findings, laboratory findings, treatment, and outcomes of patients with autoimmune GFAP‐A.

Twenty‐nine eligible patients were included, predominantly male (21/29), with acute onset in 17 patients (58.6%). Common clinical syndromes included encephalitis, meningoencephalitis, encephalomyelitis, meningoencephalomyelitis, and myelitis. Magnetic resonance imaging (MRI) of the brain revealed widespread lesions (21/28). Cerebrospinal fluid (CSF) pressure, CSF nucleated cell count, CSF protein levels, CSF chloride, serum thyroid dysfunction, and abnormal blood cytokine levels correlated with disease severity but were not associated with prognosis. There was no correlation between CSF glucose level, serum GFAP antibody titer, CSF GFAP antibody titer, ferritin levels, human herpesvirus 4 (HHV‐4) in the CSF, and disease severity or prognosis. No malignancies were detected in any patient before or after disease onset. Most patients (25/29) had favorable outcomes. Immunotherapy was effective for both the short‐ and long‐term prognosis of GFAP‐associated disease. Non‐pulse steroid therapy and pulse steroid therapy showed comparable efficacy, while monoclonal antibody therapy was also potentially effective for GFAP‐A.

A retrospective analysis of clinical features, treatment, and outcome of 29 patients with autoimmune glial fibrillary acidic protein astrocytopathy (GFAP‐A). Cerebrospinal fluid (CSF) pressure, CSF nucleated cell count, CSF protein levels, CSF chloride, serum thyroid dysfunction, and abnormal blood cytokine levels correlated with disease severity but were not associated with prognosis. There was no correlation between CSF glucose level, serum GFAP antibody titer, CSF GFAP antibody titer, ferritin levels, human herpesvirus 4 (HHV‐4) in the CSF, and disease severity or prognosis. Most patients (25/29) had favorable outcomes. Immunotherapy was effective for both the short‐term and long‐term prognosis in GFAP‐A. Non‐pulse steroid therapy and pulse steroid therapy showed comparable efficacy, while monoclonal antibody therapy was also potentially effective.