Tocilizumab Treatment in Refractory Autoimmune Glial Fibrillary Acidic Protein Astrocytopathy: A Case Report and...

Although most patients with autoimmune glial fibrillary acidic protein astrocytopathy (GFAP-A) respond to first-line immunotherapy, a subset is refractory to conventional treatments and experiences poor outcomes. This study evaluates the efficacy of tocilizumab in such refractory cases, based on clinical experience and literature review. We retrospectively analyzed a 17-year-old female with GFAP-A who presented with fever, headache, coma, language and emotional disturbances, and motor deficits. Brain magnetic resonance imaging showed T2/FLAIR hyperintensity and swelling in the bilateral basal ganglia and left hippocampus. Anti-glial fibrillary acidic protein (GFAP) antibody was positive in both serum and cerebrospinal fluid. The patient was refractory to high-dose corticosteroids, intravenous immunoglobulin, with only a transient response to rituximab. However, after five infusions of tocilizumab, she demonstrated significant improvement in language, motor function, mood, and remained in stable remission during follow-up, with no severe adverse events. A literature review identified a previously reported case of GFAP-A coexisting with anti-N-methyl-D-aspartate receptor encephalitis that similarly showed only a partial response to first-line immunotherapies before achieving marked clinical improvement after five cycles of tocilizumab. These findings suggest that tocilizumab may represent an effective therapeutic option for refractory GFAP-A, significantly improving cognitive and neuropsychiatric outcomes.