乌司奴单克隆抗体治疗儿童难治性克罗恩病六例分析并文献复习  

Ustekinumab treatment in children with refractory Crohn's disease:six cases and literature review

作　　者：梅天璐 郭姝[1] 叶晓琳 李东丹[2] 吴捷 Mei Tianlu;Guo Shu;Ye Xiaolin;Li Dongdan;Wu Jie(Department of Gastroenterology,Beijing Children's Hospital,Capital Medical University,National Center for Children's Health,Beijing 100045,China;Department of Clinical Nutrition,Beijing Children's Hospital,Capital Medical University,National Center for Children's Health,Beijing 100045,China)

机构地区：[1]国家儿童医学中心,首都医科大学附属北京儿童医院消化科,北京100045 [2]国家儿童医学中心,首都医科大学附属北京儿童医院临床营养科,北京100045

出　　处：《中华炎性肠病杂志（中英文）》2024年第3期223-229,共7页Chinese Journal of Inflammatory Bowel Diseases

摘　　要：目的探讨乌司奴单克隆抗体(UST)治疗儿童难治性克罗恩病(CD)疗效及安全性。方法回顾性分析2021年8月至2023年1月于首都医科大学附属北京儿童医院消化科使用UST治疗的6例难治性CD及CD样表现单基因病患儿的诊治过程。通过检索万方数据库、中国知网、PubMed和Medline中截止至2023年1月1日发表的UST治疗儿童CD文献,并进行相关文献复习及总结。结果纳入6例难治性CD及CD样表现单基因病患儿,均为男性。其中1例确诊为IL-10RA受体缺陷,1例确诊为IPEX综合征,1例确诊为X连锁淋巴组织增生综合征-2型。2例患儿使用UST单药治疗,3例使用UST联合沙利度胺治疗至临床缓解,1例使用UST联合英夫利西单克隆抗体双靶治疗。6例患儿经UST治疗24周均临床应答,其中5例临床缓解,随访24周,4例复查结肠镜黏膜愈合,均未发生不良事件。共检索到16篇文献。结论UST治疗可能改善儿童难治性CD临床结局。Objective To explore the efficacy and safety of ustekinumab(UST)in the treatment of refractory Crohn's disease(CD)in children.Methods The clinical features,diagnosis and treatment of 6 patients with children refractory CD and CD-like monogenic inflammatory bowel disease(IBD)who were treated with ustekinumab(UST)at the Department of Gastroenterology,Beijing Children's Hospital affiliated to Capital Medical University from August 2021 to January 2023 were retrospectively analyzed.The relevant literatures were reviewed and summarized by searching the articles on the treatment of refractory CD in children with UST published in Wanfang database,CNKI,PubMed and Medline.Results All the six children with refractory CD and CD-like monogenic IBD were boys.Among them,1 case was diagnosed as IL-10RA receptor defect,1 case was diagnosed as IPEX syndrome,and 1 case diagnosed as X-linked lymphoproliferative syndrome(XLP-2).Two patients were treated with UST only,three patients were treated with UST combined with thalidomide until presented clinical remission,and one patient was treated with UST in combination with IFX.Six children were treated with UST for over 24 weeks,and all of them presented clinical response.After treatment for more than 24 weeks,4 cases presented endoscopic response.None of the 6 patients had special adverse drug reactions during follow-up for 24 weeks.A total of 16 literatures were retrieved.Conclusion UST may induce and maintain remission in children with refractory CD.

关 键 词：克罗恩病 儿童 乌司奴单克隆抗体 难治性 生物制剂 

分 类 号：R725.7[医药卫生—儿科]