Pediatric Oncology Department, Hospital Niño Jesús, Av. Menéndez Pelayo, 65, 28009, Madrid, Spain.
Pediatric Oncology Department, Hospital Sant Joan de Déu, Barcelona, Spain.
J Neurooncol. 2018 Mar;137(1):205-213. doi: 10.1007/s11060-017-2713-4. Epub 2017 Dec 16.
Non-medulloblastoma CNS embryonal tumors (former PNET/Pineoblastomas) are aggressive malignancies with poor outcome that have been historically treated with medulloblastoma protocols. The purpose of this study is to present a tumor-specific, real-world data cohort of patients with CNS-PNET/PB to analyze quality indicators that can be implemented to improve the outcome of these patients. Patients 0-21 years with CNS-PNET treated in eight large institutions were included. Baseline characteristics, treatment and outcome [progression-free and overall survival (PFS and OS respectively)] were analyzed. From 2005 to 2014, 43 patients fulfilled entry criteria. Median age at diagnosis was 3.6 years (range 0.0-14.7). Histology was pineoblastoma (9%), ependymoblastoma (5%), ETANTR (7%) and PNET (77%). Median duration of the main symptom was 2 weeks (range 0-12). At diagnosis, 28% presented with metastatic disease. Seventeen different protocols were used on frontline treatment; 44% had gross total resection, 42% craniospinal radiotherapy, 86% chemotherapy, and 33% autologous hematopoietic stem cell transplantation (aHSCT). Median follow-up for survivors was 3.5 years (range 1.7-9.3). 3-year PFS was 31.9% (95% CI 17-47%) and OS 35.1% (95% CI 20-50%). Age, extent of resection and radiotherapy were prognostic of PFS and OS in univariate analysis (p < 0.05). Our series shows a dismal outcome for CNS-PNET, especially when compared to patients included in clinical trials. Establishing a common national strategy, implementing referral circuits and collaboration networks, and incorporating new molecular knowledge into routine clinical practice are accessible measures that can improve the outcome of these patients.
中枢神经系统胚胎性肿瘤（原 PNET/松果体母细胞瘤）是非神经母细胞瘤性中枢神经系统胚胎性肿瘤（former PNET/Pineoblastomas），是一种侵袭性恶性肿瘤，预后不良，历史上一直采用神经母细胞瘤方案进行治疗。本研究的目的是报告一组特定于肿瘤的、真实世界的 CNS-PNET/PB 患者队列，以分析可实施的质量指标，以改善这些患者的预后。纳入在 8 家大型机构接受 CNS-PNET 治疗的 0-21 岁患者。分析了基线特征、治疗和结局（无进展生存和总生存（分别为 PFS 和 OS））。2005 年至 2014 年，共有 43 例患者符合入组标准。中位诊断年龄为 3.6 岁（范围 0.0-14.7 岁）。组织学类型为松果体细胞瘤（9%）、室管膜母细胞瘤（5%）、ETANTR（7%）和 PNET（77%）。主要症状的中位持续时间为 2 周（范围 0-12 周）。诊断时，28%的患者存在转移性疾病。一线治疗中使用了 17 种不同的方案；44%患者行肿瘤全切除，42%患者行颅脊髓放疗，86%患者行化疗，33%患者行自体造血干细胞移植（aHSCT）。幸存者的中位随访时间为 3.5 年（范围 1.7-9.3 年）。3 年 PFS 为 31.9%（95%CI 17-47%），OS 为 35.1%（95%CI 20-50%）。单因素分析显示，年龄、肿瘤切除程度和放疗与 PFS 和 OS 相关（p<0.05）。本研究结果显示 CNS-PNET 的预后较差，尤其是与临床试验中纳入的患者相比。建立共同的国家策略、实施转诊回路和合作网络，以及将新的分子知识纳入常规临床实践，是改善这些患者预后的可行措施。
