目的:非典型畸胎样/横纹肌样瘤(ATRTs)是儿童时期影响中枢神经系统(CNS)的恶性胚胎性肿瘤。我们的目标是确定哪些因素,包括患者年龄,切除范围(EOR),远端转移的存在和辅助治疗的使用,影响在该单一中心治疗的非典型畸胎样/横纹肌样瘤(ATRTs)患儿的总生存率。
方法:对组织学诊断为ATRT治疗21年(1999-2020年)的患者进行回顾性队列回顾。人口统计数据,肿瘤位置,转移的存在,使用辅助治疗,切除范围(EOR),并发症,手术后的神经系统结果,收集总生存率。进行Kaplan-Meier生存分析。
结果:共有45名儿童(平均年龄2岁)接受了64次手术。25例患者年龄<1岁。15例获得了大体全切除(GTR)前辅助治疗,15例获得了近全切除(NTR),9例获得了次全切除(STR),6例获得了活检。大多数儿童术后神经系统预后良好(GOS为28/45)。14名患者存活超过4年。生存分析显示,中位生存期存在显着差异,有利于GTR和局部疾病。<1岁与>1岁之间的中位生存期没有显着差异(p=0.84)。
结论:我们发现转移是ATRT患者生存不良的重要因素。GTR,在可能的情况下,在ATRT中可能会带来显着的生存益处。年龄<1岁的儿童似乎和年龄>1岁的儿童一样,因此仍然应该考虑进行根治性手术。
Atypical teratoid/rhabdoid tumours (ATRTs) are malignant embryonal tumours of childhood that affect the central nervous system (CNS). We aim to determine which factors, including patient age, extent of resection (EOR), presence of distal metastasis and use of adjuvant therapies, affect overall survival in children with atypical teratoid/rhabdoid tumours (ATRTs) treated at this single centre.
Retrospective cohort review of patients with histological diagnosis of ATRT treated over 21 years (1999-2020) was conducted. Data on demographics, tumour location, presence of metastasis, use of adjuvant therapy, extent of resection (EOR), complications, neurological outcome post-surgery, and overall survival were collected. Kaplan-Meier survival analysis was performed.
A total of 45 children (mean age 2 years) underwent 64 operations. 25 patients were <1 year of age. Gross-total resection (GTR) pre-adjuvant therapy was achieved in 15, near-total resection (NTR) in 15, subtotal resection (STR) in 9, and biopsy in 6 children. Most children had good neurological outcomes post-operatively (28/45 with GOS 5). Fourteen patients survived longer than 4 years. Survival analysis showed a significant difference in median survival in favour of GTR and localised disease. There was no significant difference in median survival between patients <1 year vs >1 year of age (p=0.84).
We find that presence of metastasis was an important factor in poor survival in patients with ATRT. GTR, where possible, may confer significant survival benefit in ATRT. Children aged <1 year appear to have performed as well as those >1 year and therefore should still be considered for radical surgery.