Background

Metastatic atypical teratoid/rhabdoid tumors (AT/RTs) are aggressive central nervous system tumors that present during infancy and are associated with dismal outcomes. Patients receive multimodal treatment including surgical resection, systemic chemotherapy, and one or more of intrathecal chemotherapy (IT), marrow-ablative chemotherapy with autologous hematopoietic cell rescue (AuHCR) and radiation therapy (XRT). While data regarding treatment modalities for AT/RT patients exist, no comprehensive data have been published regarding the metastatic patients.

Methods

We performed a meta-analysis of 1578 articles published through September 2018, including 44 studies with a total of 123 subjects. In addition, seven patients were included through chart review of patients treated at Nationwide Children’s Hospital.

Results

Analysis of 130 patients revealed a 3-year overall survival (OS) of 25%. Age at diagnosis had a significant effect on survival (P = 0.0355); 3-year OS for infants less than 18 months was 21%, 18 to 36 months was 26%, and greater than 36 months was 36%. Location of the primary tumor, metastatic stage, and extent of surgical resection did not have a significant impact on OS. On univariate analysis, XRT (P < 0.0001), IT (P = 0.01), and AuHCR (P < 0.0001) were found to significantly improve survival. The most substantial effect was noted in patients who received AuHCR (3-year OS of 60% vs 9% in those who did not). On multivariable analysis, XRT (P = 0.0006), IT (P = 0.0124), and AuHCR (P < 0.0001) were independently associated with reduced risk of death.

Conclusions

Although more research is warranted to make generalizable conclusions, these results suggest that treatment regimens for patients with metastatic AT/RTs should include AuHCR, XRT, and IT.

中文翻译：

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荟萃分析的儿童转移性非典型畸形/大戟类肿瘤的治疗方式。

背景

转移性非典型性类畸形/类胡萝卜素肿瘤（AT / RTs）是侵袭性中枢神经系统肿瘤，在婴儿期出现，并与转归不良有关。患者接受多模式治疗，包括手术切除，全身化疗以及鞘内化疗（IT），自体造血细胞抢救（AuHCR）和放射疗法（XRT）的骨髓消融化疗。尽管存在有关AT / RT患者治疗方式的数据，但尚未发表有关转移患者的全面数据。

方法

我们对截至2018年9月发表的1578篇文章进行了荟萃分析，包括44项研究，涉及123个主题。此外，通过对全国儿童医院接受治疗的患者的病历表进行回顾，纳入了7例患者。

结果

对130位患者的分析显示，其3年总生存率（OS）为25％。诊断时的年龄对生存有重要影响（P  = 0.0355）；小于18个月的婴儿的3年OS为21％，18到36个月的婴儿为26％，大于36个月的婴儿为36％。原发肿瘤的位置，转移阶段和手术切除的范围对OS没有重大影响。在单变量分析中，发现XRT（P <0.0001），IT（P  = 0.01）和AuHCR（P <0.0001）可以显着提高生存率。在接受AuHCR的患者中观察到最明显的效果（3年OS为60％，而未接受AuHCR的患者为9％）。在多变量分析中，XRT（P  = 0.0006），IT（P  = 0.0124）和AuHCR（P <0.0001）与降低的死亡风险独立相关。

结论

尽管需要进行更多的研究才能得出可概括的结论，但这些结果表明，转移性AT / RT患者的治疗方案应包括AuHCR，XRT和IT。