Abstract

Purpose

The optimal treatment strategy for pediatric atypical teratoid rhabdoid tumor (ATRT) is inconclusive. This study evaluated the prognostic value of early radiotherapy (RT) and high-dose chemotherapy with autologous stem cell rescue (HDC/ASCR) in pediatric ATRT.

Methods

This pooled analysis included ATRT patients treated at our institution and from other studies who were identified by a search of the PubMed electronic database. The effect of patient demographics and treatment profiles on progression-free survival (PFS) and overall survival (OS) were analyzed using Cox regression.

Results

Overall, 34 patients from our institution and 436 patients from 35 published studies were included. In multivariable analysis, patients with gross total resection (GTR), early RT (time to RT interval < 2 months), and HDC/ASCR had both better PFS [hazard ratio (HR) 0.46, p\(<\) 0.001; HR 0.64, p = 0.011; and HR 0.51, p = 0.005, respectively] and OS (HR 0.55, p = 0.002; HR 0.48, p = 0.004; and HR 0.42, p < 0.001, respectively). For patients aged < 3 years, both RT and HDC/ASCR were significant favorable factors for PFS (HR 0.32 and 0.46, respectively) and OS (HR 0.40 and 0.36, respectively), while early RT was not prognostic. For patients aged ≥ 3 years, early RT was significantly associated with better PFS (HR 0.51) and HDC/ASCR did not affect PFS, and neither was related to OS.

Conclusion

Both early RT initiation and HDC/ASCR were important components in the treatment of pediatric ATRT. However, the optimal treatment strategies might differ by age.

中文翻译：

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早期放疗和大剂量化疗对不同年龄段儿童非典型性类畸胎瘤样横纹肌瘤预后的影响。

摘要

目的

小儿非典型性类畸胎瘤样横纹肌瘤（ATRT）的最佳治疗策略尚无定论。这项研究评估了早期放疗（RT）和大剂量化疗加自体干细胞抢救（HDC / ASCR）在儿科ATRT中的预后价值。

方法

该汇总分析包括在我们机构接受治疗的ATRT患者以及通过搜索PubMed电子数据库而确定的其他研究。使用Cox回归分析了患者人口统计学和治疗概况对无进展生存期（PFS）和总生存期（OS）的影响。

结果

总体而言，纳入了来自我们机构的34位患者和来自35项已发表研究的436位患者。在多变量分析中，总切除（GTR），早期RT（RT间隔<2个月）和HDC / ASCR患者均具有更好的PFS [危险比（HR）为0.46，p < 0.001）；HR 0.64，p  = 0.011；和HR 0.51，p  = 0.005]和OS（HR 0.55，p  = 0.002； HR 0.48，p  = 0.004； HR 0.42，p 分别<0.001）。对于3岁以下的患者，RT和HDC / ASCR都是PFS（分别为HR 0.32和0.46）和OS（分别为HR 0.40和0.36）的重要有利因素，而早期RT不能预后。对于≥3岁的患者，早期放疗与更好的PFS有显着相关性（HR 0.51），HDC / ASCR并不影响PFS，也与OS无关。

结论

早期放疗和HDC / ASCR都是治疗小儿ATRT的重要组成部分。但是，最佳治疗策略可能因年龄而异。