椎管内延伸的儿童神经母细胞瘤的临床特征及预后

Clinical features and prognosis of neuroblastoma with intraspinal extension in children

目的分析椎管内延伸的神经母细胞瘤(NB)患儿的临床特征、复发风险和预后影响因素,并监测、随访其长期健康问题。方法回顾性分析2007年1月至2019年12月在上海交通大学医学院附属新华医院诊治的22例椎管内延伸的NB患儿的性别、初诊年龄、临床分期和危险度分组等临床资料。将22例患儿分为非复发组(17例)和复发组(5例),采用Kaplan-Meier方法对2组患儿进行生存分析。结果1.15例(68.2%)患儿以下肢疼痛、无力、活动受限等运动神经系统症状为首发表现,其中10例(45.5%)患儿表现为中重度神经功能受压。2.非复发组绝大多数患儿神经元烯醇化酶(NSE)<200μg/L、乳酸脱氢酶(LDH)≤500 U/L、尿香草基苦杏仁酸(VMA)多处于正常范围或累及单部位椎体。3.复发组绝大多数患儿肿瘤未能完全切除、NSE≥200μg/L、LDH>500 U/L、VMA高于正常值3倍以上或存在多部位椎体转移。4.非复发组患儿中位生存期(MST)为119.4个月,复发组患儿中位MST仅25.3个月。非复发组3年总生存(OS)率为(95.5±6.4)%,复发组患儿3年OS率仅为(20.0±17.9)%(χ^(2)=9.387,P=0.002)。非复发组3年无事件生存(EFS)率为(94.1±5.7)%,复发组患儿3年EFS率仅为(20.0±17.9)%(χ^(2)=29.700,P<0.001)。5.22例椎管内延伸的NB患儿中有11例存在长期健康问题,其中运动神经功能缺陷和脊柱侧弯最为突出。结论椎管内延伸的NB患儿初次诊断时多表现为运动神经系统症状,且以中重度神经功能受压为主。椎管内肿瘤残余,较高的NSE、LDH或VMA水平以及出现多部位椎体转移的椎管内延伸的NB患儿容易复发,一旦复发预后极差。椎管内延伸的NB患儿的预后与初始神经功能受损的严重程度、肿瘤的压迫持续时间以及治疗方案相关;早期诊断与干预可降低患儿长期健康问题的风险。

Objective To explore the clinical features,risks of recurrence and prognosis of neuroblastoma(NB)with intraspinal extension in children,and to perform a long-term follow-up to monitor their health problems.Methods Clinical data of 22 children with NB with intraspinal extension treated in Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine from January 2007 to December 2019 were retrospectively analyzed,including gender,age at diagnosis,clinical stage,and risks of recurrence.They were divided into non-recurrent group and recurrent group,and the survival was analyzed by Kaplan-Meier method.Results(1)Fifteen(68.2%)children had motor nervous symptoms as the initial symptoms,including lower limb pain,weakness,and limited mobility,and 10(45.5%)showed moderate-to-severe nervous compression.(2)Neuronal enolase(NSE)(<200μg/L),lactate dehydrogenase(LDH)(≤500 U/L)and vanillylmandelic acid(VMA)were in the normal range in most of children in the non-recurrence group,and a single site was involved.(3)Tumor lesions were not completely resected in the majority of children in the recurrent group,and their NSE(≥200μg/L),LDH(>500 U/L)and VMA(more than 3 times higher)were abnormally higher,or suffered multisite pyramidal metastasis.(4)The median survival time(MST)was 119.4 months for children in the non-recurrent group,while it was only 25.3 months for those in the recurrent group.The 3-year overall survival(OS)rate was(95.5±6.4)%for the non-recurrent group,compared to only(20.0±17.9)%for children in the recurrent group(χ^(2)=9.387,P=0.002).Likewise,the 3-year event-free survival(EFS)rate for children in the non-recurrent group was(94.1±5.7)%,whereas it was only(20.0±17.9)%for children in the recurrent group(χ^(2)=29.700,P<0.001).(5)Eleven of 22 children had long-term health problems,especially motor nerve function defects and scoliosis.Conclusions Motor neurological,and moderate-to-severe neurological compression predominates are the main symptoms of NB children with intraspinal extension at the initial diagnosis.Patients who exhibit intradural tumor remnants,higher NSE,LDH,or VMA levels,and intradural extension with multiple sites of cone metastases are prone to recurrence.Once relapsed,the prognosis is extremely poor.The prognosis of NB with intraspinal extension in children is related to the severity of the initial neurological impairment,the duration of tumor compression,and the treatment regimen.Early diagnosis and intervention may reduce the risk of long-term health problems in children.