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ObjectiveTo explore the clinical characteristics and treatment outcomes of children with central nervous system (CNS) involvement in eosinophilic granulomatosis with polyangiitis (EGPA).MethodsA child who presented with EGPA complicated by CNS involvement was admitted to our hospital in June 2023. The clinical features were analyzed retrospectively, and relevant literatures were reviewed to provide a comprehensive overview of this condition.ResultsA ten-year-old girl, who had a history of recurrent cough and asthma accompanied by peripheral blood eosinophilia for eight months, was admitted to our hospital. On admission, spotted papules were visible on her hands and feet, bilateral pulmonary rales were audible. The laboratory examination revealed that the proportion of eosinophils (EOS) exceeded 10% of white blood cells, the anti-neutrophil cytoplasmic antibody (MPO-ANCA) was positive, the immunoglobulin G level was 15.80g/L, and the immunoglobulin E level was greater than 2500.00IU/mL. The imaging examination showed multiple patchy and nodular high-density shadows in both lungs as well as sinusitis. Pulmonary function tests indicated moderate ventilation and diffusion dysfunction. Bone marrow cytology demonstrated a significant increase in the proportion of eosinophils. Skin pathology confirmed leukocytoclastic vasculitis. During the hospitalization, the child had a convulsion. The magnetic resonance imaging (MRI) scan of the brain showed multiple abnormal signal shadows in the bilateral cerebral cortex and the electroencephalogram (EEG) showed epileptic waves. Following the administration of methylprednisolone pulse therapy in combination with cyclophosphamide treatment, her cough and asthma resolved, the skin rash disappeared without any further convulsions. We found that only a young EGPA patient with CNS involvement had been previously reported. The previously reported case began with long-term fever, weight loss, and purpuric rash. Both patients responded well to treatment with glucocorticoids and cyclophosphamide, experiencing significant improvement in their clinical symptoms and normalization of their peripheral blood eosinophils.ConclusionThe diagnosis of EGPA in children can be challenging. When a child is affected by EGPA, it is essential to remain vigilant for signs of CNS involvement. The treatment with glucocorticoids and cyclophosphamide is effective in managing EGPA in children.

ObjectiveThis study aimed to investigate the efficacy and safety of belimumab for treating children with refractory childhood-onset systemic lupus erythematosus (cSLE).MethodsTwenty-six cSLE patients who received belimumab treatment in our hospital from January 2020 to September 2021 (23 of them for more than 52 weeks) were enrolled in this study. Their clinical and laboratory data, assessment of disease activity, glucocorticoid dosage, and treatment-emergent adverse events (TEAEs) were retrieved for analysis. The paired samples t-test and the nonparametric test were used to compare the baseline and post-treatment data.ResultsThe mean age of onset was 10.3 ± 2.4 years old; the mean disease duration was 41.6 ± 37.4 months; the median Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score was 10 (P25, P75: 3, 17); and the mean Physician’s Global Assessment (PGA) score at baseline was 1.9 ± 1.0. Compared with the baseline values, there was a significant decrease in the 24-h urine protein quantifications at 24 and 52 weeks of treatment (P

BackgroundNeuromyelitis optica spectrum disorders (NMOSDs) are attack-relapsing autoimmune inflammatory diseases of the central nervous system, which are characterized by the presence of serological aquaporin-4 (AQP4) antibody. However, this disorder is uncommon in children, and AQP4 antibody was often found to be seronegative. However, some pediatric patients diagnosed with NMOSDs were tested to be positive for myelin oligodendrocyte glycoprotein (MOG) antibody. The previous investigations of pediatric NMOSDs were usually focused on the clinical presentation, treatment responses, and long-term prognoses, but little is known about the risk factors predicting NMOSD relapse attacks in a shorter time, especially, for Chinese children.MethodsWe retrospectively identified 64 Chinese pediatric patients, including 39 positive for AQP4 antibody, 12 positive for MOG antibody, and the rest negative for AQP4 and MOG antibodies. Independent risk factors predicting relapse in 1-year follow-up were extracted by multivariate regression analysis to establish a risk score model, its performance evaluation was analyzed using receiver operating characteristic (ROC) curve, and the independent risk factors related to relapse manifestation were also explored through multivariate logistic analysis. A nomogram was generated to assess relapse attacks in 1-year follow-up. Thirty-five patients from 3 other centers formed an external cohort to validate this nomogram.ResultsFour independent relapsed factors included discharge Expanded Disability Status Scale (EDSS) (p = 0.017), mixed-lesion onset (p = 0.010), counts (≧1) of concomitant autoantibodies (p = 0.015), and maintenance therapy (tapering steroid with mycophenolate mofetil (MMF), p = 0.009; tapering steroid with acetazolamide (AZA), p = 0.045; and tapering steroid only, p = 0.025). The risk score modeled with these four factors was correlated with the likelihood of relapse in the primary cohort (AUC of 0.912) and the validation cohort (AUC of 0.846). Also, our nomogram exhibited accurate relapse estimate in the primary cohort, the validation cohort, and the whole cohort, but also in the cohorts with positive/negative AQP4 antibody, and noticeably, it performed predictive risk improvement better than other factors in the concordance index (C-index), net reclassification improvement (NRI), and integrated discrimination improvement (IDI).ConclusionsThe risk score and nomogram could facilitate accurate prognosis of relapse risk in 1-year follow-up for pediatric NMOSDs and help clinicians provide personalized treatment to decrease the chance of relapse.