单中心116例婴幼儿神经母细胞瘤临床特征及预后分析

Clinical characteristics and prognostic analysis of 116 infants with neuroblastoma in a single center

目的分析≤18个月龄神经母细胞瘤(NB)婴幼儿的临床特征及影响预后因素。方法回顾性分析2007年1月-2017年6月在北京儿童医院血液肿瘤中心诊断并治疗的年龄≤18个月龄婴幼儿NB患儿的临床资料,总结临床特征、评估预后。应用SPSS 19.0软件进行数据分析。结果2007年1月-2017年6月共治疗116例≤18个月龄NB婴幼儿,男64例,女52例,中位年龄10(1～18)个月,随访中位时间19. 5(1～88)个月。单因素分析显示,INSS分期4期、高危、伴有骨骼或骨髓转移、MYCN基因扩增、初诊时血清乳酸脱氢酶(LDH)>500 IU/L的患儿预后不良(P <0. 05)。存在MYCN基因扩增10例,INSS分期均为3期或4期,LDH、神经元特异性烯醇化酶(NSE)明显高于无MYCN扩增病例,差异均有显著性(P=0. 028,P=0. 002,P=0. 001)。死亡病例13例,其中9例因肿瘤复发或进展死亡。116例NB婴幼儿3年无事件生存率(EFS) 83.6%,总生存率(OS) 86. 7%;5年EFS 76. 7%,OS 86. 7%。年龄≤12个月与年龄12～18个月患儿3年OS分别为90%、80. 7%,差异无显著性(P=0. 101)。INSS分期4期患儿3年OS 74. 4%、高危患儿3年OS 57. 1%、伴有MYCN基因扩增患儿NB 3年OS 26.7%,明显低于中早期、低中危、无MYCN基因扩增患儿(P=0.013,P=0.000,P=0.000)。结论≤18个月NB患儿预后良好,MYCN基因扩增、晚期病例预后差,主要死亡原因是疾病复发或进展。

Objective To analyze clinical characteristics and prognosis of 116 infants neuroblastoma.Methods The clinical data of children younger than 18 months old with neuroblastoma who were diagnosed and treated at the hematology-oncology center of Beijing Children′s Hospital(BCH)from January 2007 to June 2017 was retrospectively analyzed.Their clinical features were summarized and the prognosis was evaluated.The SPSS 19.0 statistical package was used for data analysis.Results From January 2007 to June 2017,116 patients less than 18 months old with neuroblastoma were treated,including 64 males and 52 females.The median age was 10 months old(1~18 months old).The median time of follow-up was 19.5 months(1~88 months).The univariate analysis showed that stage 4,high risk,bone or bone marrow metastasis,MYCN gene amplification,and the serum lactate dehydrogenase(LDH)﹥500 IU/L were poor prognostic factors(P﹤0.05).There were 10 cases with MYCN gene amplification.Their INSS stages were all in stage 3 or 4.The infants with MYCN gene amplification showed significantly higher LDH and neuron specific enolase(NSE)than those without MYCN gene amplification.There were statistical differences(P=0.028,P=0.002,P=0.001).There were 13 deaths,of which 9 cases died of tumor relapse or progression.The 3-year event-free survival(EFS)of 116 infants with neuroblastoma was 83.6%and the overall survival(OS)was 86.7%.The 5-year EFS was 76.7%and OS was 86.7%.The 3-year OS of infants less than 12 months old was 90%and those with 12~18 months old was 80.7%.There was no significant difference(P=0.101)between them.The 3-year OS in patients with stage 4,high risk,and MYCN gene amplification were 74.4%,57.1%and 26.7%respectively.They were significantly lower than the earlier stage,low and intermediate risk groups and without MYCN gene amplification(P=0.013,P=0.000,P=0.000).Conclusions The prognosis of infants younger than 18 months old with neuroblastoma is good.The prognosis is poor in patients with MYCN gene amplification or advance stage.The main cause of death in infants with neuroblastoma is disease relapse or progression.