Case Report: Efficacy of efgartigimod for autonomic dysfunction in patients with autoimmune glial fibrillary acidic protein astrocytopathy: a case series of 3 patients

Introduction Efgartigimod can rapidly reduce serum IgG antibody levels and decrease antibody-mediated neuroinflammation and damage. It is primarily used to treat autoimmune diseases. Glial fibrillary acidic protein astrocytopathy (GFAP-A) is a disease of the central nervous system characterized by GFAP antibodies. It has a high incidence of autonomic dysfunction in patients and there is currently no effective treatment plan. It is not known whether efgartigimod can improve autonomic dysfunction in GFAP-A. Case presentation All three patients with GFAP-A presented with autonomic dysfunction, which manifested as urinary and defecatory disorders. Marked remission of corticosteroid-refractory autonomic dysfunction was observed following combined treatment with efgartigimod and corticosteroids. All three patients presented with autonomic dysfunction. Patient 1 experienced marked improvement in autonomic function after the addition of efgartigimod, despite showing no significant improvement after high-dose pulse steroid therapy. Patient 2 showed no significant improvement after treatment with low-dose steroids, but achieved complete recovery of autonomic dysfunction after efgartigimod treatment. Patient 3 initially received low-dose steroids, with no significant improvement in autonomic dysfunction. After switching to a high dose of steroids combined with efgartigimod, the patient’s autonomic dysfunction gradually improved. Conclusion We observed that after combined treatment with efgartigimod and corticosteroids, the corticosteroid-refractory autonomic dysfunction in GFAP-A patients was significantly alleviated, providing a new clinical treatment option. However, this article is based on a small sample size and requires further validation through larger-scale studies.