Prasad Maya, Sengupta Duhita, Gollamudi Venkata Ramamohan, Parambil Badira Cheriyalinkal, Chatterjee Abhishek, Dasgupta Archya, Sahu Arpita, Sahay Ayushi, Shetty Prakash, Singh Vikas, Moiyadi Aliasgar, Gupta Tejpal, Epari Sridhar, Chinnaswamy Girish
Division of Paediatric Oncology, Tata Memorial Centre, Mumbai, India.
Homi Bhabha National Institute (HBNI), Mumbai, India.
Cancer Med. 2025 Aug;14(15):e71128. doi: 10.1002/cam4.71128.
Management of infants and young children with embryonal tumors of the central nervous system remains a challenge. We report the outcomes in these children with the strategy of surgery, pre-irradiation chemotherapy, and delayed radiation.
Children less than 3 years and diagnosed with medulloblastoma(MB), atypical teratoid rhabdoid tumor(ATRT), embryonal tumor with multilayered rosettes(ETMR), embryonal tumor tumors NOS(ET NOS) and pineoblastoma(PB), underwent standard evaluation and staging. They were treated with maximal safe surgical excision followed by pre-irradiation chemotherapy (cyclophosphamide, etoposide and carboplatin) up to 36 months of age or till disease progression, followed by either craniospinal (standard 35Gy or reduced-dose 23.4 Gy) with boost to a total of 54.8 Gy or focal irradiation depending on attained age, residual tumor, and metastasis.
72 children between January 2011 and December 2022 were eligible for inclusion; medulloblastoma-43 (59.7%), ATRT-9 (12.9%), ET NOS-8 (11.1%), ETMR-8 (11.1%) and PB-4 (5.6%). The median chemotherapy cycles were 6 (range 2-15); 41 children went on to receive RT. For the entire cohort, 3-year event-free survival (EFS) and overall survival (OS) were 39.8% ± 5.8% and 44.8% ± 5.9%. For medulloblastoma, 3-year PFS and OS were 57.4% ± 7.6% and 62% ± 7.5%. Multivariable analysis for survival in medulloblastoma found group 3 metastatic (HR 3.5, 95% CI 1.16-10.99, p = 0.026) and postoperative residual tumor (HR 1.9%, 95% CI 0.52-5.31, p = 0.24) to be significant prognostic factors. Among long-term survivors (n = 27), over 80% have late toxicities requiring intervention.
The use of moderate-intensity pre-irradiation chemotherapy is safe and effective in infants and young children with CNS ETs. Despite inferior outcomes compared to high-dose chemotherapy and intraventricular chemotherapy, this strategy could be used in resource-limited settings.
中枢神经系统胚胎性肿瘤的婴幼儿管理仍然是一项挑战。我们报告了采用手术、放疗前化疗和延迟放疗策略治疗这些儿童的结果。
年龄小于3岁且被诊断为髓母细胞瘤（MB）、非典型畸胎样横纹肌样瘤（ATRT）、具有多层菊形团的胚胎性肿瘤（ETMR）、未分类胚胎性肿瘤（ET NOS）和成松果体细胞瘤（PB）的儿童接受了标准评估和分期。他们接受了最大安全限度的手术切除，随后进行放疗前化疗（环磷酰胺、依托泊苷和卡铂），直至36个月龄或直至疾病进展，然后根据年龄、残留肿瘤和转移情况，进行全脑全脊髓放疗（标准剂量35Gy或低剂量23.4Gy）并加量至总剂量54.8Gy，或局部放疗。
2011年1月至2022年12月期间，72名儿童符合纳入标准；髓母细胞瘤43例（59.7%），ATRT 9例（12.9%），ET NOS 8例（11.1%），ETMR 8例（11.1%），PB 4例（5.6%）。化疗周期中位数为6个（范围2 - 15个）；41名儿童继续接受放疗。对于整个队列，3年无事件生存率（EFS）和总生存率（OS）分别为39.8%±5.8%和44.8%±5.9%。对于髓母细胞瘤患者，3年无进展生存率（PFS）和总生存率分别为57.4%±7.6%和62%±7.5%。髓母细胞瘤生存情况的多变量分析发现，3组转移（风险比3.5，95%置信区间1.16 - 10.99，p = 0.026）和术后残留肿瘤（风险比1.9%，95%置信区间0.52 - 5.31，p = 0.24）是显著的预后因素。在长期存活者（n = 27）中，超过80%有需要干预的晚期毒性反应。
对于患有中枢神经系统胚胎性肿瘤的婴幼儿，采用中等强度的放疗前化疗是安全有效的。尽管与高剂量化疗和脑室内化疗相比效果较差，但该策略可用于资源有限的环境。
