Autoimmune GFAP Astrocytopathy Clinical Trial
Official title:
GFAP Auto-immunity : a French Cohort Study
Glial fibrillary acidic protein (GFAP)-Immunoglobulin G (IgG) have recently been described as
a biomarker of a novel inflammatory central nervous system (CNS) disorder, termed autoimmune
GFAP astrocytopathy. Thus far, four major clinical series have been published (two from Mayo
Clinic USA, one from Italy and one from China). GFAP-IgG detected in serum or in
cerebrospinal fluid, by tissue-based assay and confirmed by cell-based assay, are associated
with encephalitis or meningoencephalitis of acute or subacute onset, less frequently with
myelitis or optic disk edema. The characteristic MRI feature is brain linear perivascular
radial gadolinium enhancement in the white matter perpendicular to the ventricle, consistent
with the immunohistochemical staining pattern of GFAP in rodent brain sections. Approximately
20% of reported cases are associated with a neoplasm (ovarian teratoma mostly). Coexisting
neural autoantibodies are described in some patients, N-methyl-D-aspartate (NMDA)-receptor
(R)-IgG mostly, followed by aquaporin 4 (AQP4)-IgG. The disease is usually corticosteroid
responsive although relapse can occur. In contrast, Chinese patients display poorer outcomes.
Pathophysiology is not well understood but the intracellular antigen location makes GFAP-IgG
unlikely pathogenic whereas animal models and neuropathologic data suggest a T-cell
immune-mediated disorder.
The aim of the investigators is to report the first French cohort of patients GFAP-IgG
positive. Investigators retrospectively assessed clinical, immunological and radiological
features, treatment response and outcomes.
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