小儿质子/光子联合登记处（PPCR）中关于非典型畸胎样/横纹肌样瘤（ATRT）的放射治疗。

Radiotherapy for Atypical Teratoid/Rhabdoid Tumor (ATRT) on the Pediatric Proton/Photon Consortium Registry (PPCR).

作者信息

Roehrig Andrew, Indelicato Daniel J, Paulino Arnold C, Ermoian Ralph, Hartsell William, Perentesis John, Hill-Kayser Christine, Lee Jae Y, Laack Nadia N, Mangona Victor, MacEwan Iain, Eaton Bree R, Gallotto Sara, Bajaj Benjamin V M, Aridgides Paul D, Yock Torunn I

机构信息

SUNY Upstate Medical University, 750 E Adams St, Syracuse, NY, 13210, USA.

University of Florida, Gainesville, USA.

出版信息

J Neurooncol. 2023 Apr;162(2):353-362. doi: 10.1007/s11060-023-04296-5. Epub 2023 Mar 23.

DOI:10.1007/s11060-023-04296-5

PMID:36951945

Abstract

PURPOSE

Atypical teratoid/rhabdoid tumors (ATRT) of the central nervous system (CNS) are rare tumors with a poor prognosis and variable use of either focal or craniospinal (CSI) radiotherapy (RT). Outcomes on the prospective Pediatric Proton/Photon Consortium Registry (PPCR) were evaluated according to RT delivered.

METHODS

Pediatric patients receiving RT were prospectively enrolled on PPCR to collect initial patient, disease, and treatment factors as well as provide follow-up for patient outcomes. All ATRT patients with evaluable data were included. Kaplan-Meier analyses with log-rank p-values and cox proportional hazards regression were performed.

RESULTS

The PPCR ATRT cohort includes 68 evaluable ATRT patients (median age 2.6 years, range 0.71-15.40) from 2012 to 2021. Median follow-up was 40.8 months (range 3.4-107.7). Treatment included surgery (65% initial gross total resection or GTR), chemotherapy (60% with myeloablative therapy including stem cell rescue) and RT. For patients with M0 stage (n = 60), 50 (83%) had focal RT and 10 (17%) had CSI. Among patients with M + stage (n = 8), 3 had focal RT and 5 had CSI. Four-year overall survival (OS, n = 68) was 56% with no differences observed between M0 and M + stage patients (p = 0.848). Local Control (LC) at 4 years did not show a difference for lower primary dose (50-53.9 Gy) compared to ≥ 54 Gy (73.3% vs 74.7%, p = 0.83). For patients with M0 disease, four-year OS for focal RT was 54.6% and for CSI was 60% (Hazard Ratio 1.04, p = 0.95. Four-year event free survival (EFS) among M0 patients for focal RT was 45.6% and for CSI was 60% (Hazard Ratio 0.71, p = 0.519). For all patients, the 4-year OS comparing focal RT with CSI was 54.4% vs 60% respectively (p = 0.944), and the 4-year EFS for focal RT or CSI was 42.8% vs 51.4% respectively (p = 0.610).

CONCLUSION

The PPCR ATRT cohort found no differences in outcomes according to receipt of either higher primary dose or larger RT field (CSI). However, most patients were M0 and received focal RT. A lower primary dose (50.4 Gy), regardless of patient age, is appealing for further study as part of multi-modality therapy.

摘要

目的

中枢神经系统（CNS）非典型畸胎样/横纹肌样瘤（ATRT）是罕见肿瘤，预后较差，局部或全脑脊髓（CSI）放疗（RT）的使用情况不一。根据所接受的放疗，对前瞻性儿科质子/光子联合登记处（PPCR）的结果进行了评估。

方法

接受放疗的儿科患者前瞻性纳入PPCR，以收集初始患者、疾病和治疗因素，并对患者预后进行随访。纳入所有具有可评估数据的ATRT患者。进行了带有对数秩p值的Kaplan-Meier分析和cox比例风险回归分析。

结果

PPCR的ATRT队列包括2012年至2021年的68例可评估的ATRT患者（中位年龄2.6岁，范围0.71 - 15.40岁）。中位随访时间为40.8个月（范围3.4 - 107.7个月）。治疗包括手术（65%为初始肉眼全切或GTR）、化疗（60%采用清髓性治疗包括干细胞救援）和放疗。对于M0期患者（n = 60），50例（83%）接受局部放疗，10例（17%）接受全脑脊髓放疗。在M +期患者（n = 8）中，3例接受局部放疗，5例接受全脑脊髓放疗。四年总生存率（OS，n = 68）为56%，M0期和M +期患者之间未观察到差异（p = 0.848）。四年局部控制率（LC）在较低初始剂量（50 - 53.9 Gy）与≥54 Gy之间无差异（73.3%对74.7%，p = 0.83）。对于M0期疾病患者，局部放疗的四年总生存率为54.6%，全脑脊髓放疗为60%（风险比1.04，p = 0.95）。M0期患者中局部放疗的四年无事件生存率（EFS）为45.6%，全脑脊髓放疗为60%（风险比0.71，p = 0.519）。对于所有患者，比较局部放疗与全脑脊髓放疗的四年总生存率分别为54.4%对60%（p = 0.944），局部放疗或全脑脊髓放疗的四年无事件生存率分别为42.8%对51.4%（p = 0.610）。