Department of Neurosurgery, The Johns Hopkins University School of Medicine, 1800 Orleans Street, Baltimore, MD, 21287, USA.
Department of Neurosurgery, Indiana University School of Medicine, Indianapolis, IN, USA.
Childs Nerv Syst. 2022 Jul;38(7):1297-1306. doi: 10.1007/s00381-022-05511-w. Epub 2022 Apr 1.
Atypical teratoid/rhabdoid tumors (AT/RTs) are malignant central nervous system (CNS) neoplasms of the young. Our study analyzed a large AT/RT cohort from the National Cancer Database (NCDB) to elucidate predictors of short-term mortality and overall survival (OS).
Information was collected on patients with histologically confirmed AT/RT using the NCDB (2004-2016). Kaplan-Meier analysis indicated OS. Prognostic factors for 30-day mortality, 90-day mortality, and OS were determined via multivariate Cox proportional hazards (CPH) and logistic regression models.
Our cohort of 189 patients had a median age of 1 year (IQR [1, 4]) and tumor size of 4.7 ± 2.0 cm at diagnosis. Seventy-two percent were under 3 years old; 55.6% were male and 71.0% were Caucasian. Fifty (27.2%) patients received only surgery (S) (OS = 5.91 months), 51 (27.7%) received surgery and chemotherapy (S + CT) (OS = 11.2 months), and 9 (4.89%) received surgery and radiotherapy (S + RT) (OS = 10.3 months). Forty-five (24.5%) received S + CT + RT combination therapy (OS = 45.4 months), 13 (17.1%) received S + CT + BMT/SCT (bone marrow or stem cell transplant) (OS = 55.5 months), and 16 (8.70%) received S + CT + RT + BMT/SCT (OS = 68.4 months). Bivariate analysis of dichotomized age (HR = 0.550, 95% CI [0.357, 0.847], p = 0.0067) demonstrated significantly increased patient survival if diagnosed at or above 1 year old. On multivariate analysis, administration of S + CT + RT, S + CT + BMT/SCT, or S + CT + RT + BMT/SCT combination therapy predicted significantly (p < 0.05) increased OS compared to surgery alone.
AT/RTs are CNS tumors where those diagnosed under 1 year old have a significantly worse prognosis. Our study demonstrates that while traditional CT, RT, and BMT/SCT combination regimens prolong life, overall survival in this population is still low.
非典型畸胎样/横纹肌样肿瘤（AT/RTs）是一种发生于年轻人群的恶性中枢神经系统（CNS）肿瘤。本研究通过国家癌症数据库（NCDB）分析了一组大型 AT/RT 队列，以阐明短期死亡率和总生存期（OS）的预测因素。
使用 NCDB（2004-2016 年）收集经组织学证实的 AT/RT 患者的信息。Kaplan-Meier 分析表明 OS。通过多变量 Cox 比例风险（CPH）和逻辑回归模型确定 30 天死亡率、90 天死亡率和 OS 的预后因素。
我们的 189 名患者队列的中位年龄为 1 岁（IQR [1, 4]），诊断时肿瘤大小为 4.7±2.0cm。72%的患者年龄小于 3 岁；55.6%为男性，71.0%为白种人。50 名患者（27.2%）仅接受手术（S）治疗（OS=5.91 个月），51 名患者（27.7%）接受手术联合化疗（S+CT）治疗（OS=11.2 个月），9 名患者（4.89%）接受手术联合放疗（S+RT）治疗（OS=10.3 个月）。45 名患者（24.5%）接受 S+CT+RT 联合治疗（OS=45.4 个月），13 名患者（17.1%）接受 S+CT+BMT/SCT 治疗（OS=55.5 个月），16 名患者（8.70%）接受 S+CT+RT+BMT/SCT 治疗（OS=68.4 个月）。对年龄进行二分类的二元分析（HR=0.550，95%CI [0.357, 0.847]，p=0.0067）表明，如果诊断年龄在 1 岁及以上，则患者的生存率显著提高。多变量分析表明，接受 S+CT+RT、S+CT+BMT/SCT 或 S+CT+RT+BMT/SCT 联合治疗的患者的 OS 显著（p<0.05）延长，与单独手术相比。
AT/RTs 是中枢神经系统肿瘤，1 岁以下诊断的患者预后明显更差。本研究表明，虽然传统的 CT、RT 和 BMT/SCT 联合方案可以延长生命，但该人群的总体生存率仍然较低。
