摘要
目的分析儿童朗格汉斯细胞组织细胞增生症(Langerhans cell histiocytosis,LCH)的临床特点、疗效和预后。方法对72例初发LCH患儿进行回顾性分析。结果72例患儿中位年龄为5岁(范围:0~14岁),颅骨受累最常见(56例,78%);BRAF-V600E突变与临床特征、疗效及预后无关(P>0.05);5年总生存率91.6%±4.2%,5年无事件生存(event-free survival,EFS)率67.5%±5.8%。危险器官受累组6周化疗有效率和5年EFS率低于无危险器官受累组(P<0.05);多系统受累组、血小板≥450×10^(9)/L组5年总生存率分别低于单系统受累组和血小板<450×10^(9)/L组(P<0.05)。其中危险器官受累是影响5年EFS的独立危险因素(P<0.05)。结论颅骨是儿童LCH最常累及部位。BRAF-V600E突变与其临床特征、疗效及预后无关。血小板增高、危险器官受累、多系统受累与预后不良相关,危险器官受累为5年EFS的独立危险因素。
Objective To study the clinical characteristics,efficacy,and prognosis of pediatric Langerhans cell histiocytosis(LCH).Methods A retrospective analysis was conducted on 72 children with newly diagnosed LCH.Results The median age of the 72 children was 5 years(range:0-14 years),with skull involvement being the most common(56 cases,77.8%).The BRAF-V600E mutation was not associated with clinical characteristics,efficacy,or prognosis(P>0.05).The 5-year overall survival rate was 91.6%±4.2%,and the 5-year event-free survival(EFS)rate was 67.5%±5.8%.The 6-week chemotherapy response rate and 5-year EFS rate were lower in the risk organ involvement group compared to the no risk organ involvement group(P<0.05).The five-year overall survival rates for the group with multi-system involvement and the group with platelet count≥450×109/L were respectively lower than those for the single-system involvement group and the group with platelet count<450×109/L(P<0.05).Risk organ involvement is an independent risk factor for 5-year EFS(P<0.05).Conclusions Skull is the most commonly affected site in pediatric LCH.The BRAF-V600E mutation is not related to clinical characteristics,efficacy,or prognosis.Elevated platelet count,risk organ involvement,and multisystem involvement are associated with poor prognosis,with risk organ involvement being an independent risk factor for 5-year EFS.
作者
蒋文轩
叶芳华
肖逸心
邓文军
俞燕
杨良春
JIANG Wen-Xuan;YE Fang-Hua;XIAO Yi-Xin;DENG Wen-Jun;YU Yan;YANG Liang-Chun(Department of Pediatric Hematology,Xiangya Hospital of Central South University,Changsha 410008,China)
出处
《中国当代儿科杂志》
北大核心
2025年第5期555-562,共8页
Chinese Journal of Contemporary Pediatrics
基金
湖南省自然科学基金项目(2024JJ5598)
长沙市科技计划项目(kq2403024)。
