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

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

methodsMedical 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.

resultsTwenty-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.