Department of Pediatrics, Center for Pediatric Cancer, National Cancer Center, Goyang, Korea.
Department of Pediatrics, Yonsei University College of Medicine, Seoul, Korea.
Cancer Res Treat. 2021 Apr;53(2):378-388. doi: 10.4143/crt.2020.756. Epub 2020 Oct 28.
Atypical teratoid/rhabdoid tumor (ATRT) is a highly aggressive malignancy with peak incidence in children aged less than 3 years. Standard treatment for central nervous system ATRT in children under the age of 3 years have not been established yet. The objective of this study was to analyze characteristics and clinical outcomes of ATRT in children aged less than 3 years.
A search of medical records from seven centers was performed between January 2005 and December 2016.
Forty-three patients were enrolled. With a median follow-up of 90 months, 27 patients (64.3%) showed at least one episode of disease progression (PD). The first date of PD was at 160 days after diagnosis. The 1- and 3-year progression-free survivals (PFS) were 51.2% and 28.5%, respectively. The 1- and 3-year overall survivals were 61.9% and 38.1%, respectively. The 3-year PFS was improved from 0% in pre-2011 to 47.4% in post-2011. Excluding one patient who did not receive any further therapy after surgery, 27 patients died due to PD (n=21), treatment-related toxicity (n=5), or unknown cause (n=1). In univariate analysis, factors associated with higher 3-year PFS were no metastases, diagnosis after 2011, early adjuvant radiotherapy, and high-dose chemotherapy (HDCT). In multivariate analysis, the use of HDCT and adjuvant radiotherapy remained significant prognostic factors for PFS (both p < 0.01).
Aggressive therapy including early adjuvant radiotherapy and HDCT could be considered to improve outcomes of ATRT in children under the age of 3 years.
非典型畸胎样/横纹肌样瘤（ATRT）是一种高度侵袭性的恶性肿瘤，发病高峰在 3 岁以下儿童。3 岁以下中枢神经系统 ATRT 的标准治疗尚未建立。本研究的目的是分析 3 岁以下儿童 ATRT 的特征和临床结果。
对 2005 年 1 月至 2016 年 12 月期间 7 个中心的病历进行了检索。
共纳入 43 例患者。中位随访 90 个月，27 例（64.3%）至少出现一次疾病进展（PD）。PD 的首次出现时间为诊断后 160 天。1 年和 3 年无进展生存率（PFS）分别为 51.2%和 28.5%。1 年和 3 年总生存率分别为 61.9%和 38.1%。2011 年前的 3 年 PFS 为 0%，2011 年后为 47.4%。排除 1 例手术后未接受任何进一步治疗的患者，27 例患者因 PD（n=21）、治疗相关毒性（n=5）或未知原因（n=1）死亡。单因素分析显示，与 3 年 PFS 较高相关的因素包括无转移、2011 年后诊断、早期辅助放疗和大剂量化疗（HDCT）。多因素分析显示，HDCT 和辅助放疗的应用是 PFS 的独立预后因素（均 p<0.01）。
包括早期辅助放疗和 HDCT 的强化治疗可考虑用于改善 3 岁以下儿童 ATRT 的结局。
