Turco Emanuela Claudia, Gnazzo Martina, Giordani Sara, Pisanò Giulia, Baldini Valentina, Giroldini Elena, Piccolo Benedetta, Neglia Cosimo, Esposito Susanna, Pera Maria Carmela
Child Neuropsychiatry Unit, Department of Medicine and Surgery, University of Parma, 43124 Parma, Italy.
Department of Biomedical, Metabolic and Neural Sciences, University of Modena and Reggio Emilia, 41121 Emilia-Romagna, Italy.
Children (Basel). 2025 Jul 24;12(8):975. doi: 10.3390/children12080975.
: Pediatric acquired demyelinating syndromes (ADSs) encompass a heterogeneous group of disorders, including multiple sclerosis (MS), MOG antibody-associated disease (MOGAD), and neuromyelitis optica spectrum disorder (NMOSD), with distinct clinical trajectories and prognoses. While analyzed collectively at baseline to reflect real-world diagnostic uncertainty, outcome predictors were also examined according to final diagnosis. Identifying early predictors is crucial for optimizing long-term outcomes. : We retrospectively analyzed 30 pediatric patients (mean onset age: 11.3 years) with ADSs. Clinical, radiological, CSF, antibody, and neurophysiological data were collected and analyzed alongside treatment strategies. Outcomes-EDSS scores, neuroradiological changes, and clinical status-were evaluated over a 3-year period. : Final diagnoses included MOGAD (36.6%), MS (33.3%), NMOSD (6.6%), ADEM (10%), and other ADSs (13.3%). At onset, ≥3 brain lesions were present in 76.7% of patients. Disease-modifying therapies (DMTs) were used in 37% and acute immunotherapy in 90%. EDSS progression was significantly associated with DMT use at multiple timepoints, with additional predictors including MRI lesion type, CSF findings, antibody status, and evoked potentials. At 3 years, neurocognitive function predicted clinical outcome. : Early immunotherapy and baseline instrumental findings are key predictors of outcome in pediatric ADSs. MOGAD showed a more favorable course, while MS and NMOSD were associated with greater long-term disability. A comprehensive, early diagnostic approach is essential for improving prognosis.
小儿获得性脱髓鞘综合征(ADSs)包括一组异质性疾病,其中包括多发性硬化症(MS)、MOG抗体相关疾病(MOGAD)和视神经脊髓炎谱系障碍(NMOSD),它们具有不同的临床病程和预后。虽然在基线时进行综合分析以反映现实世界中的诊断不确定性,但也根据最终诊断对结局预测因素进行了研究。确定早期预测因素对于优化长期结局至关重要。我们回顾性分析了30例患有ADSs的小儿患者(平均发病年龄:11.3岁)。收集并分析了临床、放射学、脑脊液、抗体和神经生理学数据以及治疗策略。在3年期间评估结局——扩展残疾状态量表(EDSS)评分、神经放射学变化和临床状态。最终诊断包括MOGAD(36.6%)、MS(33.3%)、NMOSD(6.6%)、急性播散性脑脊髓炎(ADEM,10%)和其他ADSs(13.3%)。发病时,76.7%的患者存在≥3个脑病变。37%的患者使用了疾病修饰疗法(DMTs),90%的患者使用了急性免疫疗法。EDSS进展在多个时间点与DMT的使用显著相关,其他预测因素包括MRI病变类型、脑脊液检查结果、抗体状态和诱发电位。在3年时,神经认知功能可预测临床结局。早期免疫疗法和基线检查结果是小儿ADSs结局的关键预测因素。MOGAD的病程更有利,而MS和NMOSD与更大的长期残疾相关。全面的早期诊断方法对于改善预后至关重要。