El-Hemaly Ahmed, Samir Marwa, Taha Hala, Refaat Amal, Maher Eslam, El-Beltagy Mohamed, Zaghloul Mohamed S, El-Haddad Alaa
Department of Pediatric Oncology, National Cancer Institute, Cairo University, 11765 Cairo, Egypt.
Department of Pediatric Oncology, Children's Cancer Hospital of Egypt, 12556 Cairo, Egypt.
Oncol Lett. 2024 Jan 30;27(3):129. doi: 10.3892/ol.2024.14263. eCollection 2024 Mar.
Atypical teratoid rhabdoid tumor (ATRT) is a rare type of potentially fatal childhood brain tumor. The present study aimed to examine the overall survival (OS) and event-free survival (EFS) outcomes of pediatric patients with ATRT and to analyze the impact of different prognostic factors, including age, sex, tumor site and size, metastatic disease, the extent of resection, radiotherapy, and chemotherapy, on survival. The present study included 47 patients with ATRT treated at the Children's Cancer Hospital of Egypt (Cairo, Egypt) between July 2007 and December 2017. These patients were treated according to the Dana-Farber Cancer Institute protocol 02-294 for 51 weeks. Various prognostic factors, including age, sex, tumor size and initial metastatic status, exhibited no impact on the radiological response measured at 6 weeks and at the end of treatment. The primary tumor site significantly affected the response to treatment at 6 weeks (P=0.008). Toxicity-related mortality occurred in 29.8% of patients. The median duration of the treatment protocol was 66.9 weeks. The duration of treatment was in the present cohort was longer than the actual 51 weeks of the protocol due to prolonged supportive care of the included patients. Patients who encountered toxicity received reduced dose of chemotherapy in the subsequent cycles in the protocol. Age, initial metastatic status, tumor site and resection extent did not significantly affect the patient outcomes. Preoperative tumor size significantly affected the EFS (P=0.03) and OS (P=0.04). Radiotherapy administration significantly affected the OS (P<0.001) and EFS (P<0.001). The median EFS and OS of patients were 9.3 and 10.3 months, respectively. A total of 24 (51.1%) patients exhibited disease progression or recurrence. The progression sites were local (n=6), metastatic (n=9) or both local and metastatic (n=9). The results of the present study demonstrated that the therapeutic regimen should be patient-adjusted to maintain the treatment intensity and avoid toxicity-related mortality. In lower middle-income countries, short and intensified induction followed by consolidation of treatment, either by single or tandem autologous stem cell transplant, is needed to avoid prolonged exposure to myelosuppression and toxicity-related mortality.
非典型畸胎样横纹肌样瘤（ATRT）是一种罕见的、可能致命的儿童脑肿瘤。本研究旨在探讨ATRT患儿的总生存期（OS）和无事件生存期（EFS），并分析不同预后因素，包括年龄、性别、肿瘤部位和大小、转移性疾病、切除范围、放疗和化疗对生存的影响。本研究纳入了2007年7月至2017年12月期间在埃及开罗儿童癌症医院接受治疗的47例ATRT患者。这些患者按照达纳-法伯癌症研究所02-294方案接受了51周的治疗。包括年龄、性别、肿瘤大小和初始转移状态在内的各种预后因素，对6周和治疗结束时测量的放射学反应均无影响。原发肿瘤部位对6周时的治疗反应有显著影响（P=0.008）。29.8%的患者发生了与毒性相关的死亡。治疗方案的中位持续时间为66.9周。由于对纳入患者的支持治疗时间延长，本队列中的治疗持续时间长于方案实际规定的51周。遇到毒性反应的患者在方案后续周期中接受了减量化疗。年龄、初始转移状态、肿瘤部位和切除范围对患者预后无显著影响。术前肿瘤大小对EFS（P=0.03）和OS（P=0.04）有显著影响。放疗的实施对OS（P<0.001）和EFS（P<0.001）有显著影响。患者的中位EFS和OS分别为9.3个月和10.3个月。共有24例（51.1%）患者出现疾病进展或复发。进展部位为局部（n=6）、转移（n=9）或局部和转移均有（n=9）。本研究结果表明，治疗方案应根据患者情况进行调整，以维持治疗强度并避免与毒性相关的死亡。在中低收入国家，需要进行短期强化诱导治疗，随后通过单次或串联自体干细胞移植进行巩固治疗，以避免长期暴露于骨髓抑制和与毒性相关的死亡。
