多系统受累朗格罕细胞组织细胞增生症35例临床分析  

Study on clinical features of children with multisystem involvement Langerhanscell histiocytosis 35 cases

作　　者：刘江华 刘炜[1] 李彦格 毛彦娜[1] LIU Jiang-hua;LIU Wei;LI Yan-ge;MAO Yan-na(Children’s Hospital Affiliated to Zhengzhou University,Henan Children’s Hospital,Zhengzhou Children’s Hospital,Zhengzhou 450000,China)

机构地区：[1]郑州大学附属儿童医院,河南省儿童医院,郑州儿童医院血液肿瘤科,郑州450000

出　　处：《医药论坛杂志》2020年第10期54-57,61,共5页Journal of Medical Forum

摘　　要：目的探讨儿童多系统受累朗格罕细胞组织细胞增生症(MS-LCH)的临床特点及预后。方法收集郑州大学附属儿童医院2014年7月—2018年7月初治的35例MS-LCH患儿的临床资料,按有无危险器官受累分为RO+组和RO-组,并检测B-RAF基因突变情况,所有患儿均行化疗,B-RAF基因突变阳性者加用靶向药达拉非尼。结果 35例患儿中,男20例,女15例,中位年龄38个月(2~122个月);伴危险器官受累组10例,无危险器官受累组25例,临床受累部位常见为骨、皮肤、耳、肝、脾、淋巴结、肺、垂体及造血系统;BRAF-V600E基因突变阴性者28例,阳性者7例,其中有2例接受靶向治疗;所有患儿均行化疗及随访, 35例患儿治疗结束时治愈8例,好转22例,稳定2例,死亡3例,中位随访时间36个月(3~62个月);接受治疗后,RO+组与RO-组患者生存曲线有差异(χ^2=5.025,Log Rank P=0.025),RO-组患者优于RO+患者,BRAF-V600E基因突变阳性患者与BRAF-V600E基因突变阴性患者生存曲线有差异(χ^2=14.084,Log Rank P=0.000),BRAF-V600E基因突变阴性患者优于BRAF-V600E基因突变阳性患者。结论伴危险器官受累、BRAF-V600E基因突变是影响MS-LCH患儿生存的主要因素,完善BRAF-V600E基因突变检测,应用靶向药物治疗,可提高疗效及改善预后。Objective To investigate the clinical features and prognosis of children with multisystem involvement Langerhanscell histiocytosis(MS-LCH).Methods The clinical data of 35 children with newly diagnosed MS-LCH from July 2014 to July 2018 in our department was collected. The patients were divided into RO+ group and RO-group according to the presence or absence of dangerous organ involvement, and the mutation of B-RAF gene was detected. All the children received chemotherapy, and those with positive mutation of B-RAF gene received targeted drug darafenib.Results Of all 35 cases, 20 were male and 15 were female,the median age were 38 months(2~122 months);25 cases were without risk organ(RO-group) and 15 were with risk organ(RO+ group). LCH usually affected skeleton system, skin, ear, liver, spleen, lymph node, pituitarium and hematopoietic system.28 cases were with negative mutation of BRAF-V600 E and 7 cases were with the positive mutation of which 2 received darafenib.All children were followed up effectively, with a median follow-up time of 36 months(3~62 months). After treatment, the survivorship curve between RO-and RO+ groups(χ^2=5.025, Log Rank P=0.025), BRAF-V600 E mutation positive and BRAF-V600 E mutation negative(χ^2=14.084, Log Rank P=0.000) were significantly different. And the RO-group was superior to RO+ group, the BRAF-V600 E mutation positive was superior to BRAF-V600 E mutation negative.Conclusion Risk organ involvement and poor treatment response are the main factors affecting the survival of MS-LCH children. The gene mutation detection of BRAF-V600 E and the application of targeted drugs and second-line therapy can improve the efficacy and prognosis.

关 键 词：多系统受累朗格罕细胞组织细胞增生症 预后 儿童 

分 类 号：R733[医药卫生—肿瘤]