Department of Neurological Surgery, University of Miami Miller School of Medicine, 1095 NW 14th Terrace, Miami, FL, 33136, USA.
Department of Neurologic Surgery, Mayo Clinic, Rochester, MN, USA.
Childs Nerv Syst. 2021 Apr;37(4):1077-1085. doi: 10.1007/s00381-020-04972-1. Epub 2020 Nov 24.
Atypical teratoid/rhabdoid tumor (ATRT) is a rare and largely pediatric diagnosis, with poor survival. Diagnosis below the age of 3 years is characteristically seen as a poor prognostic sign. However, elucidating if clinical differences exist within this niche age group has never been attempted before. Correspondingly, we sought to characterize clinical profile of ATRT diagnoses before the age of 3 years based on separate ages of diagnosis.
All pediatric ATRT patients aged < 3 years in the US National Cancer Database (NCDB) between 2005 and 2016 were retrospectively reviewed. Age groups were divided based on diagnoses at ages 0-1 years in group 1, 1-2 years in group 2, and 2-3 years in group 3. Data were summarized, and overall survival (OS) was modeled using Kaplan-Meier and Cox regression analyses.
A total of 354 ATRT diagnoses were made before the age of 3 years, with surgery used in 316 (89%) cases, chemotherapy in 242 (68%) cases, and radiation therapy in 118 (33%) cases. In terms of diagnosis age, there were 153 (43%) in group 1, 137 (39%) in group 2, and 64 (18%) in group 3. With respect to OS, median value was 9.9 months in group 1, 28.4 months in group 2, and 15.9 months in group 3. Upon multivariate analysis, receiving radiation therapy was the only parameter shared amongst all three groups as independently prognostic of longer OS (HR 0.53, P = 0.01 in group 1; HR 0.34, P < 0.01 in group 2; HR 0.31, P < 0.01 in group 3). In group 1, surgery (HR 0.47, P < 0.01) and chemotherapy (HR 0.44, P < 0.01) were also independently prognostic of longer OS. In group 3, multiple socioeconomic parameters were identified to independently predict longer OS. There were no additional predictive parameters identified in group 2.
Although ATRT diagnosed before the age of 3 is typically viewed a poor prognostic age category, our findings demonstrate that the clinical profile of this pediatric niche is highly heterogeneous based on age of diagnosis. Survival of only those diagnosed between 0 and 1 years is independently prognosticated by all three treatment modalities; patients diagnosed between 1 and 2 years trend towards longest survival, and socioeconomic parameters are most influential in those diagnosed between 2 and 3 years.
胚胎性横纹肌样肿瘤（ATRT）是一种罕见的、主要发生于儿童的疾病，预后较差。3 岁以下的诊断特征是预后不良的标志。然而，在此特定年龄段内是否存在临床差异，以前从未尝试过进行研究。相应地，我们试图根据诊断时的不同年龄，对 3 岁以下的 ATRT 患者的临床特征进行描述。
我们回顾了美国国家癌症数据库（NCDB）中 2005 年至 2016 年间年龄<3 岁的所有小儿 ATRT 患者。根据诊断时的年龄将患者分为 0-1 岁为一组（1 组）、1-2 岁为一组（2 组）、2-3 岁为一组（3 组）。总结数据，并使用 Kaplan-Meier 和 Cox 回归分析来模拟总生存期（OS）。
共有 354 例 ATRT 患者的年龄<3 岁，其中 316 例（89%）患者接受了手术，242 例（68%）患者接受了化疗，118 例（33%）患者接受了放疗。按诊断年龄，1 组 153 例（43%）、2 组 137 例（39%）、3 组 64 例（18%）。在 OS 方面，1 组的中位值为 9.9 个月，2 组为 28.4 个月，3 组为 15.9 个月。多因素分析显示，接受放疗是三组共同的独立预后因素，与更长的 OS 相关（1 组 HR 0.53，P=0.01；2 组 HR 0.34，P<0.01；3 组 HR 0.31，P<0.01）。在 1 组中，手术（HR 0.47，P<0.01）和化疗（HR 0.44，P<0.01）也是独立的预后因素。在 3 组中，确定了多个社会经济参数独立预测更长的 OS。在 2 组中未发现其他预测参数。
虽然<3 岁诊断的 ATRT 通常被认为是预后不良的年龄类别，但我们的研究结果表明，根据诊断年龄，该儿科亚组的临床特征具有高度异质性。仅诊断为 0-1 岁的患者的生存由三种治疗方式独立预测；诊断为 1-2 岁的患者有较长的生存趋势，诊断为 2-3 岁的患者的社会经济参数最具影响力。
