Clinical analysis of pediatric recurrent autoimmune glial fibrillary acidic protein astrocytopathy

Abstract

Objective To examine the clinical characteristics, treatment, and prognosis of recurrent autoimmune glial fibrillary acidic protein astrocytopathy (GFAP-A). Methods The data of 17 pediatric patients with recurrent autoimmune GFAP-A who were treated in the neurology department of Hunan Children’s Hospital from January 2015 to January 2024 were collected, and the clinical presentation, laboratory tests, neuroradiology results, electroencephalograms, treatment regimens, and prognosis of these patients were retrospectively analyzed and summarized. Results These 17 patients included 6 males and 11 females. A total of 28 recurrences occurred, with a median of 1 (1, 2) recurrence per patient. Patients experienced 1–5 symptoms on recurrence, with 3 recurrences exhibiting new symptoms. The median number of symptoms at recurrence was 1 (1, 2), with a median mRS score at recurrence of 3 (2, 4) and a median length of hospitalization of 10 (8, 16) days per recurrence episode. The mean number of symptoms, mRS, and length of hospitalization at recurrence were all lower than at initial disease onset (P < 0.05). The serum GFAP antibody positivity rate was 50% and the cerebrospinal fluid GFAP antibody positivity rate was 14.3% at recurrence. The cerebrospinal fluid abnormality rate was 28.6% at recurrence. Abnormal electroencephalograms were observed in 14 (50%) recurrences. There were 16 (57.1%) recurrences with an increased number of lesions visible on head MRI compared with pre-recurrence levels, while there were 8 (66.7%) recurrences with an increased number of lesions visible on spinal cord MRI compared with pre-recurrence levels. First-line immunotherapy was used at initial disease onset with second-line immunotherapy used only in 1 patient. Second-line immunotherapy was used in 11 recurrences. Conclusion Pediatric GFAP-A tends to recur. The condition at recurrence is milder than at initial disease onset. In most cases, the symptoms seen at recurrence are among those that occurred at initial onset, while new symptoms may occur on recurrence in a small number of patients. In addition to new symptoms and disease worsening during recurrence, abnormal antibody levels, cerebrospinal fluid routine biochemistry test results, electroencephalography results, and radiology findings may also be observed. First-line immunotherapy or long-term combined second-line immunotherapy can be used during recurrence.