全身型幼年特发性关节炎相关难治性巨噬细胞活化综合征的挽救治疗1例报告  

Salvage therapy for refractory macrophage activation syndrome associated with systemic juvenile idiopathic arthritis:a case report

作　　者：吴建强[1] 沈丹萍 卢美萍[1] WU Jianqiang;SHEN Danping;LU Meiping(Department of Rheumatology Immunology and Allergy,Children's Hospital,Zhejiang University School of Medicine,National Clinical Research Center for Child Health,Hangzhou 310000,Zhejiang,China)

机构地区：[1]浙江大学医学院附属儿童医院、国家儿童健康与疾病临床医学研究中心、风湿免疫过敏科,浙江杭州310000

出　　处：《临床儿科杂志》2025年第3期216-219,共4页Journal of Clinical Pediatrics

基　　金：浙江省“尖兵”“领雁”研发攻关计划资助(No.2023C03032)。

摘　　要：目的寻求全身型幼年特发性关节炎(sJIA)相关的难治性巨噬细胞活化综合征(MAS)的挽救治疗至关重要,本文旨探讨芦可替尼治疗难治性sJIA-MAS的应用选择和治疗效果。方法回顾性分析1例难治性sJIA-MAS患儿的临床资料以及芦可替尼应用后转归情况。结果11岁女孩诊断sJIA有4年,既往合并2次MAS,此次因sJIA活动入院并在治疗过程中再发MAS,患儿经3轮大剂量甲基波尼松龙冲击联合环孢素A、托珠单抗(TCZ)治疗后病情无好转,仍持续高热且合并严重肝功能损害等实验室指标异常。在停TCZ予加用芦可替尼并调整剂量为10 mg,bid口服后,患儿病情好转,激素顺利减量。芦可替尼治疗近3个月停用,患儿疾病无复发。结论芦可替尼可能是难治性sJIA-MAS的挽救治疗选择。Objective The pursuit of salvage treatment for refractory macrophage activation syndrome(MAS)associated with systemic juvenile idiopathic arthritis(sJIA)is of paramount importance.This paper aims to investigate the application options and therapeutic efficacy of ruxolitinib in the treatment of refractory sJIA-MAS.Methods A retrospective analysis was performed on the clinical data of a child with refractory sJIA-MAS and the outcome following the administration of ruxolitinib.Results An 11-year-old girl,diagnosed with sJIA for four years and having experienced two previous episodes of MAS,was admitted to the hospital due to active sJIA and developed MAS again during the treatment course.Despite three rounds of high-dose methylprednisolone pulse therapy in combination with cyclosporine A and tocilizumab(TCZ),her condition failed to improve,with persistent high fever and severe liver function impairment,among other abnormal laboratory indicators.After discontinuing TCZ and initiating ruxolitinib with an adjusted oral dose of 10 mg twice daily,the child's condition improved,enabling a smooth reduction of the hormone dosage.Ruxolitinib was discontinued after approximately three months of treatment,and there was no recurrence of the disease.Conclusion Ruxolitinib may potentially serve as a salvage treatment option for refractory sJIA-MAS.

关 键 词：全身型幼年特发性关节炎 巨噬细胞活化综合征 难治性 芦可替尼 

分 类 号：R72[医药卫生—儿科]