Krisch Jacquelyn M, Ermoian Ralph P, Indelicato Daniel J, Lee Jae Y, Perentesis John P, Perkins Stephanie M, Laack Nadia N, Chang John H, MacEwan Iain J, Wolden Suzanne L, Wang Dongliang, Yock Torunn I, Aridgides Paul D
Department of Radiation Oncology, SUNY Upstate Medical University, Syracuse, NY, USA.
Department of Radiation Oncology, University of Washington/ Fred Hutchinson Cancer Center, Seattle, WA, USA.
J Neurooncol. 2025 May 13. doi: 10.1007/s11060-025-05065-2.
Embryonal tumor with multilayered rosettes (ETMR) is a rare pediatric CNS embryonal tumor with poor survival. The Pediatric Proton/Photon Consortium Registry (PPCR) was queried for outcomes data from prospectively consenting pediatric patients with ETMR treated with proton radiation therapy (RT).
20 patients (2013-2021) at 9 institutions had ETMR; 2 with prior RT were excluded from statistical analyses (PPCR ETMR, N = 18). Overall Survival (OS) and Event Free Survival (EFS) analyses were performed using the Kaplan-Meier method and log-rank values. Median follow-up was calculated using the reverse Kaplan-Meier method.
Median age at RT was 3.0 years (1.7-12.2); median follow-up was 55.5 months (2.6-119.4). 8 patients (44%) expired and 6 patients (33%) are surviving ≥ 55 months. 11 (61%) patients received systemic therapy with stem cell support. The majority (89%) had focal RT (median dose 54 Gy), while 2 patients received craniospinal irradiation (CSI, 30.6-36 Gy). 4-year OS and EFS were 59.6% and 54.2%, respectively. Local control (LC) at 4 years was 81%. No differences in OS or EFS were observed for receipt of systemic therapy with stem cell support (p = 0.361, p = 0.57), progression prior to RT (p = 0.127, p = 0.18), or surgery to RT ≥ 200 days (p = 0.35, p = 0.254). Symptomatic radionecrosis was not reported.
Focal proton RT provided effective local control as part of multimodality therapy for ETMR, with encouraging survival for this rare and often infant age tumor. Outcomes for CSI were limited to 2 patients treated upfront, and 1 patient receiving salvage CSI for disseminated relapse after focal RT who is surviving > 1 year.
DFCI protocol 12-103, clinicaltrials.gov NCT01696721, date of registration 9/27/2012.
多层菊形团胚胎性肿瘤（ETMR）是一种罕见的儿童中枢神经系统胚胎性肿瘤，生存率低。查询儿科质子/光子联合登记处（PPCR），以获取前瞻性同意接受质子放射治疗（RT）的ETMR儿科患者的结局数据。
9家机构的20例患者（2013 - 2021年）患有ETMR；2例曾接受过放疗的患者被排除在统计分析之外（PPCR ETMR，N = 18）。使用Kaplan-Meier方法和对数秩值进行总生存（OS）和无事件生存（EFS）分析。使用反向Kaplan-Meier方法计算中位随访时间。
放疗时的中位年龄为3.0岁（1.7 - 12.2岁）；中位随访时间为55.5个月（2.6 - 119.4个月）。8例患者（44%）死亡，6例患者（33%）存活≥55个月。11例（61%）患者接受了有干细胞支持的全身治疗。大多数患者（89%）接受局部放疗（中位剂量54 Gy），而2例患者接受了全脑全脊髓照射（CSI，30.6 - 36 Gy）。4年总生存率和无事件生存率分别为59.6%和54.2%。4年局部控制率为81%。在接受有干细胞支持的全身治疗（p = 0.361，p = 0.57）、放疗前进展（p = 0.127，p = 0.18）或放疗前手术≥200天（p = 0.35，p = 0.254）方面，未观察到总生存率或无事件生存率的差异。未报告有症状的放射性坏死。
作为ETMR多模式治疗的一部分，局部质子放疗提供了有效的局部控制，对于这种罕见且常为婴儿期的肿瘤，生存率令人鼓舞。全脑全脊髓照射的结局仅限于2例 upfront 治疗的患者，以及1例在局部放疗后因播散性复发接受挽救性全脑全脊髓照射且存活超过1年的患者。
DFCI方案12 - 103，clinicaltrials.gov NCT01696721，注册日期2012年9月27日。
