Finkelstein-Shechter Tal, Gassas Adam, Mabbott Donald, Huang Annie, Bartels Ute, Tabori Uri, Janzen Laura, Hawkins Cynthia, Taylor Michael, Bouffet Eric
Bone Marrow Transplant Program, Hospital for Sick Children, Toronto, Canada.
J Pediatr Hematol Oncol. 2010 Jul;32(5):e182-6. doi: 10.1097/MPH.0b013e3181dce1a2.
To retrospectively review an institutional experience in managing atypical teratoid/rhabdoid tumors (AT/RT) of the Central Nervous System with high-dose chemotherapy in infants and children less than 4 years old.
MATERIALS/METHODS: Eight AT/RT patients were identified during the study period 2003 to 2008. Tumor location was supratentorial in 3 cases, infratentorial in 3 cases, and multifocal in 2 patients. Five patients presented with leptomeningeal dissemination. Two of these patients did not receive any active therapy. After surgery, the 6 remaining patients received induction therapy followed by sequential high-dose chemotherapy with autologous stem cell rescue. Two patients receive focal irradiation.
At a median follow-up of 52 months, 4 patients are alive without evidence of tumor. Three of these patients, including 2 with metastatic disease were not irradiated. However, all surviving patients exhibit neuro-cognitive impairment, either at baseline assessment or upon follow-up.
This experience confirms that a subset of young AT/RT patients may achieve long-term survival with intensive and high-dose chemotherapy. The role of radiotherapy is still unclear. However, evidence of neuro-cognitive impairment even in the absence of radiotherapy in this young population suggests that systematic introduction of radiotherapy in current protocols should be carefully assessed.
回顾性分析本机构对4岁以下婴幼儿中枢神经系统非典型畸胎样/横纹肌样瘤（AT/RT）采用大剂量化疗的治疗经验。
材料/方法：在2003年至2008年研究期间共确定8例AT/RT患者。肿瘤位于幕上3例，幕下3例，2例为多灶性。5例患者出现软脑膜播散。其中2例患者未接受任何积极治疗。手术后，其余6例患者接受诱导治疗，随后序贯进行大剂量化疗及自体干细胞解救。2例患者接受局部放疗。
中位随访52个月时，4例患者存活且无肿瘤证据。其中3例患者，包括2例有转移疾病的患者未接受放疗。然而，所有存活患者在基线评估或随访时均表现出神经认知障碍。
该经验证实，部分年轻的AT/RT患者通过强化大剂量化疗可能实现长期生存。放疗的作用仍不明确。然而，即使在该年轻人群中未进行放疗也有神经认知障碍的证据，这表明应仔细评估在当前方案中系统性引入放疗的情况。
