Department of Pediatrics, Fond. IRCCS Istituto Nazionale dei Tumori, Milano, Italy.
Pediatr Blood Cancer. 2013 Dec;60(12):2031-5. doi: 10.1002/pbc.24540. Epub 2013 Jul 13.
A protocol for the intensive treatment of non-cerebellar PNET (CNS-PNET) combining chemotherapy and radiotherapy was launched in 2000. Efforts were subsequently made to improve the prognosis and to de-escalate the treatment for selected patient groups.
Twenty-eight consecutive patients were enrolled for a high-dose drug schedule (methotrexate, etoposide, cyclophosphamide, and carboplatin ± vincristine), followed by hyperfractionated accelerated CSI (HART-CSI) at total doses of 31-39 Gy, depending on the patient's age, with two high-dose thiotepa courses following CSI. After the first 15 patients had been treated, craniospinal irradiation (CSI) was replaced with focal radiotherapy (RT) for selected cases (non-metastatic and not progressing during induction chemotherapy). Eight of the 28 children received the same chemotherapy but conventionally fractionated focal RT at 54 Gy.
The 5-year progression-free survival (PFS), event-free survival (EFS), and overall survival (OS) rates were 62%, 53%, and 52%, respectively, for the whole series, and 70%, 70%, and 87% for the eight focally irradiated children. Residual disease and metastases were not prognostically significant. In children with residual disease, response to RT was significant (5-year PFS 59% vs. 20%, P = 0.01), while the total dose of CSI was not. There were three treatment-related toxic events. Relapses were local in seven cases (including two of the eight focally irradiated patients), and both local and disseminated in 2.
This intensive schedule enabled treatment stratification for the purposes of radiation, thereby sparing some children full-dose CSI. Local control is the main goal of treatment for CNS-PNET.
2000 年启动了一项针对非小脑 PNET（CNS-PNET）的强化治疗方案，该方案结合了化疗和放疗。随后，努力改善预后并为选定的患者群体降低治疗强度。
连续入组 28 例患者，采用大剂量药物方案（甲氨蝶呤、依托泊苷、环磷酰胺和卡铂±长春新碱），随后根据患者年龄接受超分割加速全脑脊髓照射（HART-CSI），总剂量为 31-39 Gy，在 CSI 后进行两个大剂量噻替哌疗程。在前 15 例患者接受治疗后，对于选择的病例（诱导化疗期间无转移和进展），用局部放疗（RT）代替颅脊髓照射（CSI）。28 例患儿中的 8 例接受了相同的化疗，但采用常规分割局部 RT 54 Gy。
全系列的 5 年无进展生存率（PFS）、无事件生存率（EFS）和总生存率（OS）分别为 62%、53%和 52%，8 例接受局部放疗的患儿分别为 70%、70%和 87%。残留疾病和转移并非预后不良的因素。在有残留疾病的患儿中，对 RT 的反应具有显著意义（5 年 PFS 59%比 20%，P = 0.01），而 CSI 的总剂量没有显著意义。有 3 例治疗相关的毒性事件。7 例复发为局部（包括 8 例局部放疗患儿中的 2 例），2 例为局部和播散性。
这种强化治疗方案可实现放疗分层，从而使部分患儿免于接受全剂量 CSI。局部控制是 CNS-PNET 治疗的主要目标。
