儿童颅内非典型畸胎样/横纹肌样瘤的诊治及预后分析

Diagnosis,treatment and prognostic analysis of pediatric intracranial atypical teratoid/rhabdoid tumor

目的探讨儿童颅内非典型畸胎样/横纹肌样瘤(AT/RT)的诊断、治疗策略及预后。方法回顾性分析2012年1月至2019年6月郑州大学第一附属医院经病理学诊断为颅内AT/RT的15例患儿的临床资料,采用Kaplan-Meier法计算总体生存(OS)率和无进展生存(PFS)率。采用Log-rank比较不同组间生存差异,采用COX回归模型研究影响生存的因素。结果本组患儿男12例,女3例;中位年龄5.5岁(8个月~17.1岁);患儿均首选手术切除,其中全切10例,次全切5例;6例患儿行"手术+放疗+化疗+鞘内注射",4例患儿行"手术+化疗+鞘内注射",2例患儿行"手术+放疗",3例患儿仅行手术治疗。截至2020年1月,15例患儿中位生存期为18个月(1~27个月),存活率为33.3%。患儿1年OS率和PFS率分别为71.5%和49.7%,2年OS率和PFS率分别为17.9%和0。Log-rank检验结果显示<3岁和≥3岁患儿1年OS率分别为87.5%和57.1%,差异有统计学意义(χ^(2)=4.257,P=0.039)。全切组和次全切组患儿1年OS率分别为90.0%和40.0%,差异有统计学意义(χ^(2)=6.057,P=0.014)。肿瘤未播散组和播散组患儿1年OS率分别为100.0%和33.3%,差异有统计学意义(χ^(2)=9.865,P=0.002)。标危组和高危组患儿1年OS率分别为88.9%和41.7%,差异有统计学意义(χ^(2)=5.111,P=0.024)。COX回归模型显示,年龄、肿瘤切除程度、肿瘤是否播散和危险度分层是影响患儿OS预后的独立危险因素[风险比(HR)=3.411、3.795、5.245、3.397,P=0.025、0.011、0.001、0.017]。结论儿童颅内AT/RT临床罕见,初诊困难,预后差,最大范围的安全切除仍是首选治疗,年龄、肿瘤切除程度、肿瘤是否播散和危险度分层是AT/RT患儿的独立预后因素。

Objective To explore the diagnosis,treatment and prognostic of pediatric intracranial atypical teratoid/rhabdoid tumor(AT/RT).Methods A total of 15 pediatric patients with intracranial AT/RT were treated between January 2012 and June 2019 at the First Affiliated Hospital of Zhengzhou University.The clinical data were retrospectively analyzed.Overall survival(OS)rate and progression free survival(PFS)rate were calculated by adopting Kaplan-Meier method.The differences between the 2 groups were tested by performing Log-rank method,and the prognostic factors were analyzed by COX regression.Results There were 12 males and 3 females,with the median age of 5.5 years(ranging from 8 months to 17.1 years).All patients underwent surgical resection.Gross-total resection(GTR)was achieved in 10 cases and subtotal resection(STR)was carried out in 5 patients.The conducted treatments were as follows:surgery+radiotherapy+chemotherapy+intrathecal injection in 6 cases,surgery+chemotherapy+intrathecal injection in 4 cases,surgery+radiotherapy in 2 cases,and surgery alone in 3 cases.Until January 2020,the median survival time of all the 15 patients was 18 months(ranged 1-27 months),and the survival rate was 33.3%.The 1-year OS rate and PFS rate for all 15 cases were 71.5%and 49.7%,respectively.The 2-year OS rate and PFS rate were 17.9%and 0,respectively.Log-rank analyses revealed that the 1-year OS rates of children less than 3 years old and those older than 3 years were 87.5%and 57.1%,respectively(χ^(2)=6.057,P=0.014).The 1-year OS rates of children with GTR and those with STR were 90.0%and 40.0%,respectively(χ^(2)=6.057,P=0.014).The 1-year OS rates of children with tumor dissemination and those without tumor dissemination were 100.0%and 33.3%,respectively(χ^(2)=9.865,P=0.002).The 1-year OS rates of children in the standard-risk group and those in the high-risk group were 88.9%and 41.7%,respectively(χ^(2)=5.111,P=0.024).COX regression analyses proved that age,the extent of tumor resection,tumor dissemination and risk stratification are independent risk factors for prognosis[hazard radio(HR)=3.411,3.795,5.245,3.397;P=0.025,0.011,0.001,0.017].Conclusions Pediatric intracranial AT/RT is rare.The preliminary diagnosis and prognosis are difficult and poor,respectively.The complete resection of tumors with maximal safety remains the primary treatment.Age,the extent of tumor resection,tumor dissemination and risk stratification are independent prognostic factors for AT/RT children.