中剂量阿糖胞苷治疗儿童难治性高危朗格罕细胞组织细胞增生症的效果及安全性分析  

Efficacy and safety of intermediate-dose cytarabine in the treatment of children with refractory high risk Langerhans cell histiocytosis

作　　者：王雯倩 葛健 马宏浩[1] 廉红云[1] 崔蕾 张莉[1] 李志刚 王天有[1] 张蕊[1] Wang Wenqian;Ge Jian;Ma Honghao;Lian Hongyun;Cui Lei;Zhang Li;Li Zhigang;Wang Tianyou;Zhang Rui(Hematology Center,Beijing Children′s Hospital,Capital Medical University,National Center for Children′s Health,Beijing Key Laboratory of Pediatric Hematology Oncology,National Key Discipline of Pediatrics,Key Laboratory of Major Diseases in Children,Ministry of Education,Beijing 100045,China;Laboratory of Hematologic Diseases,Beijing Pediatric Research Institute,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血液病中心,儿童血液病与肿瘤分子分型北京市重点实验室,儿科学国家重点学科,儿科重大疾病研究教育部重点实验室,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,北京市儿科研究所血液疾病研究室,北京100045

出　　处：《中华儿科杂志》2023年第12期1118-1123,共6页Chinese Journal of Pediatrics

基　　金：国家自然科学基金(82070202,82141119);北京市自然科学基金(7232058);首都卫生发展科研专项(首发2020-2-2093,2020-2-1141,2022-2-1141);北京市医院管理中心儿科学科协同发展中心专项(XTZD20180201)。

摘　　要：目的:分析中剂量阿糖胞苷治疗儿童难治性危险器官受累朗格罕细胞组织细胞增生症(LCH)的效果、安全性及远期预后。方法:回顾性分析2013年1月至2016年12月北京儿童医院血液病中心收治的多系统危险器官受累、一线治疗失败且选用中剂量阿糖胞苷[250 mg/(m^(2)·次),2次/d]治疗的17例LCH患儿临床资料。患儿共接受2种治疗方案:在长春地辛和地塞米松的基础上,方案A为中剂量阿糖胞苷联合克拉屈滨,方案B为单纯中剂量阿糖胞苷。分析两种方案的治疗效果、安全性及预后。结果:17例LCH患儿中男11例、女6例,诊断年龄2.1(1.6,2.7)岁。10例患儿接受了方案A治疗,所有患儿在诱导治疗8个疗程后评估疾病好转,疾病活动评分(DAS)从治疗前的5.5(3.0,9.0)分下降至1.0(0,2.3)分。7例患儿接受了方案B治疗,6例患儿在诱导治疗8个疗程后评估疾病好转,DAS从治疗前的4.0(2.0,4.0)分下降至1.0(0,2.0)分。A组及B组患儿分别随访6.2(4.9,7.2)年及5.2(3.7,5.8)年。A组及B组患儿5年总体生存率均为100.0%,5年无事件生存率分别为(88.9±10.5)%及(85.7±13.2)%。8例A组患儿及2例B组患儿出现了3、4级骨髓抑制。结论:中剂量阿糖胞苷(联合或不联合克拉屈滨)对于难治性高危LCH患儿是一种有效且安全的化疗方案,远期预后较好。Objective To analyze the efficacy,safety,and long-term prognosis of intermediate-dose cytarabine(Ara-c)regimen in the treatment of children with refractory risk organ involvement Langerhans cell histiocytosis(LCH).Methods Clinical data of 17 children with multisystem and risk organ involvement LCH who failed the first-line therapy and were treated with intermediate-dose Ara-c(250 mg/m^(2),twice daily)regimen in the Hematology Center,Beijing Children′s Hospital from January 2013 to December 2016 were analyzed retrospectively.In addition to the basic treatment of vindesine and dexamethasone,the patients received two regimens:regimen A:the intermediate-dose Ara-c combined with cladribine and regimen B:the intermediate-dose Ara-c alone.The efficacy,safety and prognosis of the two regimens were analyzed.Results Among all 17 patients,there were 11 males and 6 females,with the diagnosis age of 2.1(1.6,2.7)years.Ten children received regimen A,all of them achieved active disease-better(AD-B)after 8 courses of induction therapy.The disease activity scores(DAS)decreased from 5.5(3.0,9.0)to 1.0(0,2.3).Seven children received regimen B,and 6 of them achieved AD-B after 8 courses of induction therapy.The DAS decreased from 4.0(2.0,4.0)to 1.0(0,2.0).The follow-up time was 6.2(4.9,7.2)and 5.2(3.7,5.8)years in group A and B.The 5-year overall survival rate was 100.0%in both groups,and the 5-year event free survival rate was(88.9±10.5)%and(85.7±13.2)%in group A and B.Grade 3 or 4 myelosuppression was observed in 8 patients in group A and 2 patients in group B.Conclusions The intermediate-dose Ara-c regimen(with or without cladribine)is effective and safe for patients with refractory high-risk LCH,with a good long-term prognosis.

关 键 词：组织细胞增多症 郎格尔汉斯细胞 儿童 阿糖胞苷 

分 类 号：R725.5[医药卫生—儿科]