中国循证儿科杂志2025，Vol.20Issue(4)：272-276,5.DOI:10.3969/j.issn.1673-5501.2025.04.006
泰它西普治疗儿童眼肌型重症肌无力4例病例系列报告
Ocular myasthenia gravis treated with telitacicept in 4 children:A case series report
摘要
Abstract
Background Children with ocular myasthenia gravis(OMG)are prone to relapse under traditional treatment with corticosteroid-related side effects,while the biological agent telitacicept has shown good efficacy in the treatment of adult MG.Objective To evaluate the efficacy and safety of telitacicept in the treatment of children with OMG.Design Case series report.Methods We conducted a retrospective study to collect the clinical data of four children with OMG who treated with telitacicept in Children's Hospital,Zhejiang University School of Medicine from April 2024 to April 2025,including clinical characteristics,scale scores,laboratory tests and follow-up information.The clinical outcomes and adverse events were also evaluated.Main out-come measures Changes in clinical symptoms and adjustments of medication.Results Among the 4 patients,2 were males and 2 were females.The median age of onset was 3.2 years(range 1.7 to 10.5 years),and the median course of disease before telit-acicept treatment was 8.2 years(range 2.7 to 12.2 years).These children were successively treated with pyridostigmine bromide,corticosteroids,and nonsteroidal immunosuppressants such as azathioprine and mycophenolate mofetil,but their symptoms of pto-sis,diplopia,and ocular duction limitation did not improve,and adverse effects associated with long-term corticosteroid use emerged.After 4 to 12 months of treatment with telitacicept,all children were able to discontinue glucocorticoids and other immu-nosuppressants.At the last follow-up,their scores of quantitative MG scale and MG-activity of daily living scale decreased com-pared to baseline,and no adverse drug reactions were reported during treatment.Conclusion Telitacicept demonstrates signifi-cant efficacy in improving clinical symptoms of children with OMG and exhibits a relatively good safety profile,which is beneficial in reducing the reliance on corticosteroids and immunosuppressants.关键词
眼肌型重症肌无力/泰它西普/儿童/生物制剂/靶向治疗Key words
Ocular myasthenia gravis/Telitacicept/Children/Biological agent/Targeted treatment引用本文复制引用
毛姗姗,颜悦,盛国霞,诸丹蕙,姜仔彦,赵聪颖,高峰..泰它西普治疗儿童眼肌型重症肌无力4例病例系列报告[J].中国循证儿科杂志,2025,20(4):272-276,5.基金项目
国家自然科学基金:82271735 （）
