Cramer Paula, Nikolaus Marc, Loos Sebastian, Denecke Jonas, Knierim Ellen, Müller Dominik, Weber Lutz T, Taylan Christina, Thumfart Julia
Department of Paediatric Nephrology, Gastroenterology and Metabolic Diseases, Charité Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health (BIH), Campus Virchow-Klinikum, Augustenburger Platz 1, 13353, Berlin, Germany.
Department of Paediatric Neurology, Charité Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health (BIH), Campus Virchow-Klinikum, Augustenburger Platz 1, 13353, Berlin, Germany; Center for Chronically Sick Children, Charité Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health (BIH), Campus Virchow-Klinikum, Augustenburger Platz 1, 13353, Berlin, Germany.
Eur J Paediatr Neurol. 2025 Jan;54:58-63. doi: 10.1016/j.ejpn.2024.12.005. Epub 2024 Dec 21.
Therapeutic apheresis (TA) are promising treatment option for neuroimmunological disorders. In paediatrics, the available data is limited, particularly for the use of IA. The aim of this study was to analyse the use of PE and IA in children and adolescents, with emphasis on outcome and neurological course after treatment as well as the safety of the two modalities.
Clinical data from paediatric patients with neuroimmunological disorders treated with TA in two German university children's hospitals between 2015 and 2022 were retrospectively analysed.
In total, 39 patients underwent 322 sessions of TA, of which 184 were IA and 138 PE. The most common diagnosis was autoimmune encephalitis in 39 % (n = 15) of the patients. Other indications were central nervous system inflammatory demyelinating disorders in 21 % (n = 8), Guillain-Barré syndrome in 18 % (n = 7), Myelin Oligodendrocyte Glycopeptide-antibody associated syndromes in 8 % (n = 3), Myasthenia gravis in 5 % (n = 2) and other neurological disorders in 10 % (n = 4). Overall, there was an improvement in 76 % of patients (81 % with IA, 70 % with PE; p = 0.41) immediately after treatment and an improvement in 88 % of patients (90 % with IA, 85 % with PE; p = 0.63) one month after treatment. Complications occurred in 13 % of all sessions (13 % with IA and 13 % with PE; p = 1). Most complications were considered as moderate.
Both, IA and PE, are effective treatment options in the therapy of neuroimmunological disorders in children and adolescents, with no major differences in terms of efficiency or safety.
治疗性血液成分单采术(TA)是治疗神经免疫性疾病的一种有前景的治疗选择。在儿科领域,可用数据有限,尤其是关于免疫吸附(IA)的使用。本研究的目的是分析儿童和青少年中血浆置换(PE)和IA的使用情况,重点关注治疗后的结局和神经病程以及这两种方式的安全性。
回顾性分析了2015年至2022年间在两家德国大学儿童医院接受TA治疗的神经免疫性疾病儿科患者的临床数据。
共有39例患者接受了322次TA治疗,其中184次为IA,138次为PE。最常见的诊断是自身免疫性脑炎,占患者的39%(n = 15)。其他适应症包括中枢神经系统炎性脱髓鞘疾病,占21%(n = 8);格林-巴利综合征,占18%(n = 7);髓鞘少突胶质细胞糖蛋白抗体相关综合征,占8%(n = 3);重症肌无力,占5%(n = 2);以及其他神经疾病,占10%(n = 4)。总体而言,76%的患者在治疗后立即有所改善(IA组为81%,PE组为70%;p = 0.41),治疗后一个月,88%的患者有所改善(IA组为90%,PE组为85%;p = 0.63)。所有治疗中有13%发生并发症(IA组和PE组均为13%;p = 1)。大多数并发症被认为是中度的。
IA和PE都是治疗儿童和青少年神经免疫性疾病的有效治疗选择,在疗效或安全性方面没有重大差异。