Department of Radiation Oncology, Beaumont Health, Royal Oak, Michigan.
Department of Pediatric Hematology/Oncology, Beaumont Health, Royal Oak, Michigan.
Pediatr Blood Cancer. 2019 Dec;66(12):e27969. doi: 10.1002/pbc.27969. Epub 2019 Aug 28.
Atypical teratoid/rhabdoid tumors (AT/RTs) are rare aggressive central nervous system tumors. The use of radiation therapy (RT) remains controversial, especially for patients younger than three years of age. The purpose of the current investigation is to robustly analyze the impact of RT among pediatric AT/RT patients using the Surveillance, Epidemiology, and End Results (SEER) database.
SEER 18 Custom Data registries were queried for AT/RT (ICD-0-3 9508/3). A total of 190 pediatric AT/RT patients were identified, of whom 102 underwent surgery + chemotherapy and 88 underwent trimodality therapy. Univariate and multivariable analyses using Kaplan-Meier and Cox proportional hazards regression modeling were performed. Propensity-score matched analysis with inverse probability of treatment weighting was performed to account for indication bias. The landmark method was used to account for immortal time bias.
The majority of patients were <3 years old (75.8%). Patients <3 were more likely to be treated without RT as compared with older patients (62% vs 38%). Doubly robust MVA identified distant disease as a negative prognostic factor (HR 2.1, P = 0.003), whereas trimodality therapy was strongly protective (HR 0.39, P < 0.001). Infants (<1), toddlers (1-2), and older children (3+) all benefited from trimodality therapy, with largest benefit for infants (HR 0.34, P = 0.02) and toddlers (HR 0.31, P < 0.001).
The current study provides further evidence that trimodality therapy improves clinical outcomes among patients with AT/RT. This finding was most pronounced for younger patients; therefore, further studies are needed to confirm this finding in this vulnerable population.
非典型畸胎样/横纹肌样瘤（AT/RTs）是一种罕见的侵袭性中枢神经系统肿瘤。放射治疗（RT）的应用仍存在争议，尤其是对于三岁以下的患者。本研究的目的是使用监测、流行病学和最终结果（SEER）数据库，对儿科 AT/RT 患者接受 RT 的影响进行强有力的分析。
通过 SEER 18 定制数据登记系统查询 AT/RT（ICD-0-3 9508/3）。共确定了 190 例儿科 AT/RT 患者，其中 102 例接受了手术+化疗，88 例接受了三联疗法。采用 Kaplan-Meier 和 Cox 比例风险回归模型进行单变量和多变量分析。采用逆概率治疗加权法进行倾向评分匹配分析，以纠正指示偏倚。采用里程碑法纠正不朽时间偏倚。
大多数患者年龄<3 岁（75.8%）。与年龄较大的患者相比，年龄<3 岁的患者更有可能不接受 RT 治疗（62%比 38%）。双重稳健的 MVA 确定远处疾病为负预后因素（HR 2.1，P=0.003），而三联疗法具有很强的保护作用（HR 0.39，P<0.001）。婴儿（<1 岁）、幼儿（1-2 岁）和大龄儿童（3 岁以上）均从三联疗法中获益，婴儿（HR 0.34，P=0.02）和幼儿（HR 0.31，P<0.001）获益最大。
本研究进一步证明三联疗法可改善 AT/RT 患者的临床结局。这一发现对年轻患者最为显著；因此，需要进一步的研究来证实这一发现。
