Central nervous system atypical teratoid/rhabdoid tumor: Results of therapy in children enrolled in a registry
Central nervous system atypical teratoid/rhabdoid tumor: Results of therapy in children enrolled in a registry
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DOI：
10.1200/jco.2004.07.073
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发表时间：
2004-07-15
影响因子：
45.3
通讯作者：
Biegel, JA
中科院分区：
文献类型：
--
作者：
Hilden, JM;Meerbaum, S;Biegel, JA
目的中枢神经系统非典型畸胎瘤样/横纹肌样瘤 (AT/RT) 是一种极其罕见且具有侵袭性的儿童早期肿瘤。传统的婴儿脑肿瘤治疗效果不佳，导致缺乏明确的治疗指南。已经建立了一个登记处来创建结果数据库并促进该肿瘤的生物学研究。材料和方法向治疗医生提供了标准化数据表，列出了将发送到登记处进行摘要的报告。每年两次寻求随访信息。结果 42 名患者的信息完整。诊断时的中位年龄为 24 个月。九名患者（21%）在诊断时患有播散性疾病。十六个肿瘤位于幕下； 26个位于幕上。 20 名患者 (48%) 接受了初次完全切除。主要治疗包括所有患者的化疗、13 名患者（31%）的放疗、13 名患者（31%）的干细胞挽救以及 16 名患者（38%）的鞘内化疗。据报道，分别有 9 名和 19 名患者出现疾病复发或进展。 27 名患者 (64%) 死于疾病（诊断后 3 至 62 个月），一名患者死于毒性。 14 名患者 (33%) 没有表现出任何疾病证据（诊断后 9.5 至 96 个月）。中位生存期为 16.75 个月，中位事件树生存期为 10 个月。 结论 积极治疗延长了部分儿童的自然病程。需要专门针对 AT/RT 设计的前瞻性多机构和国家临床试验。应继续在 AT/RT 注册表中注册。 (C) 2004 年，美国临床肿瘤学会。
PurposeAtypical teratoid/rhabdoid tumor (AT/RT) of the CNS is an extremely rare and aggressive tumor of early childhood. The poor outcome with conventional infant brain tumor therapy has resulted in a lack of clear treatment guidelines. A registry has been established to create an outcomes database and to facilitate biology studies for this tumor.Materials and MethodsA standardized data sheet was provided to treating physicians listing the reports that were to be sent to the registry for abstraction. Follow-up information was sought twice yearly.ResultsInformation was complete for 42 patients. Median age at diagnosis was 24 months. Nine patients (21%) had disseminated disease at diagnosis. Sixteen tumors were infratentorial; 26 were supratentorial. Twenty patients (48%) received a primary complete resection. Primary therapy included chemotherapy in all patients, radiotherapy in 13 patients (31%), stem-cell rescue in 13 patients (31%), and intrathecal chemotherapy in 16 patients (38%). Recurrent or progressive disease was reported in nine and 19 patients, respectively. Twenty-seven patients (64%) are dead of disease (3 to 62 months from diagnosis) and one patient died of toxicity. Fourteen patients (33%) show no evidence of disease (9.5 to 96 months from diagnosis). The median survival is 16.75 months and the median event-tree survival is 10 months.ConclusionAggressive therapy has prolonged the natural history in a subset of children. Prospective multi-institutional and national clinical trials designed specifically for AT/RT are needed. Enrollment onto the AT/RT registry should be continued. (C) 2004 by American Society of Clinical Oncology.