[Clinical characteristics and prognosis of refractory N-methyl-D-aspartate receptor antibody encephalitis in children].
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Objective: To investigate the clinical characteristics, risk factors and prognosis of children with refractory anti-N-methyl-D-aspartate (NMDA) receptor antibody encephalitis. Methods: A retrospective cohort study was conducted in 149 children diagnosed with NMDA receptor antibody encephalitis in the Department of Pediatric Neurology, XiangYa Hospital of Central South University from February 2014 to March 2024. Clinical data, including demographics, MRI features, electroencephalography (EEG), immunotherapy and prognosis were collected. Children were divided into refractory and non-refractory groups. Intergroup comparisons were performed using the χ² test or Fisher exact test, independent-samples t test or Mann-Whitney U test as appropriate. Multivariate Logistic regression model was used to identify risk factors for refractoriness. Results: Among 139 children ultimately included in the analysis, there were 53 males and 86 females, with an onset age of (8.5±4.2) years. There were 21 children in the refractory group and 118 children in the non-refractory group. The refractory group exhibited higher proportions of motor disorders, psychiatric symptoms, sleep disorders, language disorders, consciousness disorders, autonomic dysfunction, tumor comorbidity, pediatric intensive care unit admission, modified Rankin scale scores of 4-5 at onset, absent occipital dominant rhythm on EEG, epileptiform discharges on EEG, diffuse β activity EEG, requirement for plasma exchange, and B-lymphocyte percentage than the non-refractory group (all P<0.05). During a follow-up of 31 (17, 47) months, the modified Rankin scale scores of refractory group and non-refractory group at the last follow-up was 1 (0, 3) and 1 (1, 1), the difference had statistically significant (Z=2.13, P=0.033). Two children in the refractory group died, no deaths in the non-refractory group, the difference had statistically significant (χ²=11.40, P<0.01). Independent risk factors for refractoriness included autonomic dysfunction (OR=5.97, 95%CI 1.66-21.52), EEG epileptiform discharges (OR=4.91, 95%CI 1.53-15.78) and absent occipital dominant rhythm on EEG (OR=4.07, 95%CI 1.13-14.64). Conclusions: Children with refractory NMDA receptor antibody encephalitis present with severe clinical manifestations. Autonomic dysfunction, EEG epileptiform discharges and absent occipital dominant rhythm on EEG are independent risk factors for refractoriness. Early identification and intensified immunotherapy are critical for improving outcomes.