Department of Neurosurgery, Beijing Tiantan Hospital, Capital Medical University, Tiantan Xili 6, Dongcheng District, Beijing, People's Republic of China.
Neurosurg Rev. 2020 Feb;43(1):281-292. doi: 10.1007/s10143-018-1055-9. Epub 2018 Dec 8.
No standard treatment protocol to guide the management of the primary central nervous system atypical teratoid rhabdoid tumors (ATRTs). To evaluate the efficacy of GTR (gross total resection), RT (radiotherapy), CCMT (conventional chemotherapy), or intensified chemotherapy (ICMT) and verify the optimal treatment strategy. A total of 501 cases (18 cases from our center and 483 cases from published literature) were eligible for analysis. Clinical characteristics were reviewed, and overall survival (OS) of each combined treatment modality was compared. These prior publication data were processed according to PRISMA guidelines. This study included 265 (52.9%) males and 216 (43.1%) females. The median age of the cohort was 2.2 years with 295 (58.9%) cases younger than 3 years. GTR was achieved in 217 (43.3%) patients. Initial adjuvant CCMT, CCMT plus intrathecal chemotherapy (ITCMT), CCMT plus high-dose chemotherapy (HDCMT), and CCMT plus ITCMT and HDCMT were administered in 228 (45.5%), 78 (15.6%), 55 (11.0%), and 24 (4.8%) patients, respectively. Radiotherapy (RT) was prescribed in 266 (53.1%) patients. Fewer patients younger than 3 years old received RT (21.9% vs 33.0%, p < 0.001, chi-square test). The OS of the entire cohort at 1, 3, and 5 years were 56.6, 35.9, and 30.8%, respectively. After adjusting for age and sex, GTR (HR 0.630; p < 0.001), RT (HR = 0.295; p < 0.001), CCMT (HR = 0.382; p < 0.001), and ICMT (HR = 0.209; p < 0.001) were independent prognostic factors. The 3-year OS of surgery alone, surgery plus CCMT, surgery plus RT, surgery plus ICMT, surgery plus CCMT and RT, and surgery plus ICMT and RT were 8.9, 13.4, 23.7, 37.4, 48.3, and 68.5%, respectively. When taking into consideration the extent of tumor resection (n = 462), GTR followed by RT, CCMT, intrathecal chemotherapy, and high-dose chemotherapy provided the best OS (5-year OS 88.2%). In younger children, adjuvant ICMT had a greater 3-year OS than adjuvant RT alone (34.0% vs 0%, p = .001). This study identified independent favorable predictors for OS of ATRTs and distinguished significantly different OS following various treatment modalities. If tolerable, intensive treatment with GTR followed by adjuvant RT and ICMT is recommended. Intensified CCMT could be an alternative to avoid radiological radiotoxicity for younger children CRD42018098841.
目前尚无指导原发性中枢神经系统非典型畸胎样横纹肌样肿瘤（ATRTs）治疗的标准治疗方案。本研究旨在评估最大限度肿瘤切除（GTR）、放疗（RT）、常规化疗（CCMT）、强化化疗（ICMT）的疗效，并验证最佳治疗策略。共纳入 501 例患者（18 例来自本中心，483 例来自文献），对其临床特征进行回顾性分析，并比较了每种联合治疗方法的总生存率（OS）。根据 PRISMA 指南对这些先前发表的数据进行处理。本研究包括 265 例男性（52.9%）和 216 例女性（43.1%），中位年龄为 2.2 岁，其中 295 例（58.9%）年龄小于 3 岁。217 例（43.3%）患者实现了 GTR。228 例（45.5%）、78 例（15.6%）、55 例（11.0%）和 24 例（4.8%）患者分别接受了初始辅助 CCMT、CCMT 联合鞘内化疗（ICMT）、CCMT 联合大剂量化疗（HDCMT）和 CCMT 联合 ICMT 和 HDCMT。266 例（53.1%）患者接受了 RT。3 岁以下患者接受 RT 的比例较低（21.9% vs 33.0%，p<0.001，卡方检验）。全队列的 1、3 和 5 年 OS 分别为 56.6%、35.9%和 30.8%。在校正年龄和性别后，GTR（HR 0.630；p<0.001）、RT（HR=0.295；p<0.001）、CCMT（HR=0.382；p<0.001）和 ICMT（HR=0.209；p<0.001）是独立的预后因素。单纯手术、手术加 CCMT、手术加 RT、手术加 ICMT、手术加 CCMT 和 RT 以及手术加 ICMT 和 RT 的 3 年 OS 分别为 8.9%、13.4%、23.7%、37.4%、48.3%和 68.5%。考虑到肿瘤切除程度（n=462），GTR 后联合 RT、CCMT、鞘内化疗和大剂量化疗可获得最佳 OS（5 年 OS 为 88.2%）。在年龄较小的儿童中，辅助 ICMT 的 3 年 OS 优于单独辅助 RT（34.0% vs 0%，p=0.001）。本研究确定了 ATRT OS 的独立有利预测因素，并区分了不同治疗方法后的显著不同的 OS。如果可以耐受，建议采用 GTR 后辅助 RT 和 ICMT 的强化治疗。对于年龄较小的儿童，强化 CCMT 可能是避免放射性放射性毒性的替代方案。
