Division of Hematology Oncology Bone Marrow Transplantation, Alberta Children's Hospital, Calgary, Alberta, Canada.
Division of Hematology/Oncology, Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada.
Pediatr Blood Cancer. 2015 Jul;62(7):1265-9. doi: 10.1002/pbc.25441. Epub 2015 Mar 18.
Because atypical teratoid rhabdoid tumor(ATRT) is a rare disease of infancy carrying a grim prognosis, focus on long-term outcome, especially neurocognitive remained very limited. With new era of multimodality therapy, an increasing proportion of patients are now long-term survivors.
Retrospective review of neuropsychological (NP) status of survivors from the Canadian ATRT registry.
Among 77 patients diagnosed between 1995-2012, 16(22%) were survivors. Formal NP assessments were available in eight patients. Partial information on academic achievement was available on three additional patients. There were four girls and seven boys diagnosed at a median age of 27.5 months. Seven patients underwent complete resection and three had metastatic disease. All but one received sequential high dose chemotherapy. Five patients received intrathecal chemotherapy. Three patients underwent radiation. Median age at time of formal NP assessment was 7.6 years (3.9-9.8). Full Scale Intellectual Quotient (FSIQ) ranged from less than 50-119 (mean 78). Simple expressive and receptive language appeared relatively preserved. Three recently diagnosed patients (median time assessment from diagnosis 2.6 years (1.6-4.7)) had average to high average FSIQ, academic and visual spatial skills, visual, and verbal memory. Five other patients diagnosed earlier and tested at a median time of 4.9 years (3.3-8.3) post-diagnosis had a FSIQ ranging from <50 to 71. Approximately 50% of their scores were in the impaired range.
Overall this cohort appears significantly impaired at school age despite the absence of systematic radiotherapy. Larger series focusing on neurocognitive outcome are needed in the current context where treatment strategies include adjuvant radiation.
由于婴儿罕见的间叶性横纹肌样肿瘤（ATRT）预后较差，因此重点关注长期预后，特别是神经认知方面的预后，这方面的研究仍然非常有限。随着多模式治疗新时代的到来，越来越多的患者现在成为长期幸存者。
回顾性分析加拿大 ATRT 登记处幸存者的神经心理学（NP）状况。
在 1995 年至 2012 年间诊断的 77 名患者中，有 16 名（22%）为幸存者。对 8 名患者进行了正式的 NP 评估。另外 3 名患者提供了部分学业成绩信息。4 名女孩和 7 名男孩被诊断为中位年龄 27.5 个月。7 名患者接受了完全切除术，3 名患者发生了转移性疾病。除 1 名患者外，所有患者均接受了序贯高剂量化疗。5 名患者接受了鞘内化疗。3 名患者接受了放疗。正式 NP 评估的中位年龄为 7.6 岁（3.9-9.8）。全量表智商（FSIQ）范围从低于 50-119（平均 78）。简单的表达和接受语言似乎相对保留。最近诊断的 3 名患者（从诊断到评估的中位时间为 2.6 年（1.6-4.7））具有平均至高平均 FSIQ、学业和视觉空间技能、视觉和语言记忆。另外 5 名患者更早被诊断并在诊断后 4.9 年（3.3-8.3）的中位时间进行测试，FSIQ 范围从低于 50 到 71。他们大约 50%的分数处于受损范围内。
尽管没有系统的放疗，但在这个时代，治疗策略包括辅助放疗，本队列在学龄期总体上明显受损。在当前的治疗背景下，需要更大规模的系列研究来关注神经认知结果。
