不同骨骼转移部位高危神经母细胞瘤患儿的临床特征与预后  

Clinical characteristics and prognosis of high-risk neuroblastoma patients with skeletal metastases at different sites

作　　者：范洪君 黄程[1] 周宇晨[1] 朱帅 段超[1] 赵文[1] 赵倩[1] 王希思[1] 张大伟[1] 金眉[1] 马晓莉[1] 苏雁[1] Fan Hongjun;Huang Cheng;Zhou Yuchen;Zhu Shuai;Duan Chao;Zhao Wen;Zhao Qian;Wang Xisi;Zhang Dawei;Jin Mei;Ma Xiaoli;Su Yan(Department of Medical Oncology,Pediatric Oncology Center,Beijing Children′s Hospital,Capital Medical University,National Center for Children′s Health,Beijing Key Laboratory of Pediatric Hematology Oncology,Key Laboratory of Major Diseases in Children,Ministry of Education,Beijing 100045,China)

机构地区：[1]国家儿童医学中心(北京),首都医科大学附属北京儿童医院,儿童肿瘤中心肿瘤内科,北京市儿童血液肿瘤重点实验室,儿科重大疾病研究教育部重点实验室,北京100045

出　　处：《中华实用儿科临床杂志》2023年第5期331-338,共8页Chinese Journal of Applied Clinical Pediatrics

基　　金：北京市自然科学基金(7222054)。

摘　　要：目的分析伴骨骼转移高危神经母细胞瘤(HR-NB)患儿的临床特点和相关预后因素。方法回顾性分析2007年1月至2018年12月首都医科大学附属北京儿童医院肿瘤内科收治的336例初诊伴骨骼转移HR-NB患儿临床资料,采用Kaplan-Meier法进行生存分析,单因素预后分析采用Log-Rank检验,Cox模型用于多因素预后分析。结果336例患儿发病中位年龄43(4~148)月龄;男188例,女148例。面颅骨转移89例(26.5%),脑颅骨转移193例(57.4%),椎骨转移298例(88.7%),胸肋骨转移183例(54.5%),骨盆转移270例(80.4%),上肢骨182例(54.2%)和下肢骨转移240例(71.4%)。患儿5年无事件生存(EFS)率和总生存(OS)率分别为(30.4±2.7)%和(41.3±2.9)%。单因素分析显示,预后分类不良、形态学为神经母细胞瘤间质贫乏和节细胞神经母细胞瘤混合型、有丝分裂核分裂指数高、乳酸脱氢酶≥587 U/L、血清铁蛋白≥92μg/L、MYCN扩增及1p杂合性缺失及面颅骨、脑颅骨、胸肋骨、骨盆、上肢骨和下肢骨骨骼转移的HR-NB患儿5年OS率显著降低(P<0.05)。全部7部位广泛骨骼转移患儿5年OS率仅为(14.2±5.9)%,显著低于单部位及其他多部位骨骼转移患儿[5年OS率分别为(66.0±10.2)%和(43.6±3.4)%,χ^(2)=45.722,P<0.05]。Cox多因素分析显示,MYCN扩增和面颅骨骨骼转移是影响伴骨骼转移HR-NB患儿预后的独立危险因素(HR=4.165、2.560,95%CI:2.356~7.363、1.519~4.315,均P<0.05)。结论初诊时伴多部位广泛骨骼转移HR-NB患儿预后极差。MYCN扩增和面颅骨骨骼转移是影响伴骨骼转移HR-NB患儿预后的不良因素。Objective To analyze the clinical characteristics and prognostic factors of high-risk neuroblastoma(HR-NB)patients with skeletal metastasis.Methods The clinical features of 336 newly treated HR-NB patients with skeletal metastases admitted to the Department of Medical Oncology of Beijing Children′s Hospital,Capital Medical University from January 2007 to December 2018 were analyzed retrospectively.Kaplan-Meier method was used for the survival analysis,and Log-Rank test was used for univariate prognosis analysis.The Cox model was used to analyze the multifactorial prognostic analysis.Results A total of 336 HR-NB patients were recruited,involving 188 males and 148 females with the median age of onset of at 43(4-148)months.Skeletal metastases affected the viscerocranium(89 cases,26.5%),neurocranium(193 cases,57.4%),vertebrae(298 cases,88.7%),sternum and ribs(183 cases,54.5%),pelvis(270 cases,80.4%),upper limbs(182 cases,54.2%)and lower limbs(240 cases,71.4%).The 5-year event-free survival(EFS)rate and overall survival(OS)rate were(30.4±2.7)%and(41.3±2.9)%,respectively.Univariate analysis showed a significantly lower 5-year OS rate in skeletal metastatic HR-NB patients with poor prognostic classification,the morphology of neuroblastoma(stroma-poor)and ganglioneuroblastoma(intermixed),high index of mitosis-karyorrhexis index,lactate dehydrogenase≥587 U/L,serum ferritin≥92μg/L,MYCN amplification and 1p loss of heterozygosity,and metastases in the viscerocranium,neurocranium,vertebrae,sternum and ribs,pelvis,upper limbs and lower limbs(all P<0.05).The 5-year OS rate of HR-NB patients with all 7 regions of skeletal metastases was only(14.2±5.9)%,which was significantly lower than that in patients with a single region metastasis or multi-region metastases[(66.0±10.2)%vs.(43.6±3.4)%,χ^(2)=45.722,P<0.05].Cox multifactorial analysis showed that MYCN amplification(HR=4.165,95%CI:2.356-7.363)and the viscerocranium metastasis(HR=2.560,95%CI:1.519-4.315)were the independent risk factors affecting the prognosis of HR

关 键 词：神经母细胞瘤 高危 骨骼转移 预后 

分 类 号：R739.4[医药卫生—肿瘤]