Metwally Sarah Magdy, El-Ayadi Moatasem, Maher Eslam, El-Minawi Mohamed Sherif, Zaghloul Mohamed S, Taha Hala, Aboulnaga Sherif, Sidhom Iman
Department of Pediatric Oncology, National Cancer Institute, Cairo University, Cairo, Egypt.
Department of Pediatric Oncology, Children's Cancer Hospital Egypt (CCHE-57357), Cairo, Egypt.
J Neurooncol. 2025 Jun 10. doi: 10.1007/s11060-025-05098-7.
Medulloblastoma (MB) treatment includes surgery, irradiation, and chemotherapy (CT). Cisplatin-based regimens for standard-risk (SR) MB are effective but associated with significant toxicities, particularly ototoxicity. This study compares the toxicity profiles of two CT regimens, focusing on grade ≥ 3 ototoxicity, hematologic, hepatic, renal, and neurologic toxicities.
This study included SR-MB patients aged 3-18 years. Cohort A (2016-2019) received adjuvant CT adopted from the Children's Oncology Group (COG) A9961 Regimen A protocol, with data collected retrospectively. Cohort B (2020-July 2022) received CT adopted from the ACNS0331 protocol, with data collected prospectively. Toxicities were assessed and graded using the Common Terminology Criteria for Adverse Events (CTCAE) v5.0.
A total of 168 patients aged 3 to 18 years were enrolled, 112 (67%) in cohort A and 56 (33%) in cohort B. Grade ≥ 3 ototoxicity was significantly higher in cohort A (24% vs. 3.6%, p < 0.001). Neurotoxicity occurred in 26% vs. 12.5% (p = 0.046). Anemia and thrombocytopenia in 71% vs. 52% (p = 0.04). Febrile neutropenia was more common in cohort B (66% vs. 38%, p < 0.001). No significant differences were found in grade ≥ 3 leukopenia, nephrotoxicity or hepatotoxicity. The 2-year overall survival was 96.4% (95% CI: 93.1-99.9) in cohort A vs. 86.6% (95% CI: 77.8-96.4) in cohort B (p = 0.11). Event-free survival was 92.9% (95% CI: 88.2-97.8) vs. 86.8% (95% CI: 78-96.4) (p = 0.29).
Partial substitution of cisplatin with cyclophosphamide showed a better toxicity profile, particularly for ototoxicity and neurotoxicity, with no significant difference in survival.
髓母细胞瘤（MB）的治疗包括手术、放疗和化疗（CT）。基于顺铂的标准风险（SR）MB治疗方案有效，但伴有显著毒性，尤其是耳毒性。本研究比较了两种化疗方案的毒性特征，重点关注≥3级耳毒性、血液学、肝脏、肾脏和神经毒性。
本研究纳入了3至18岁的SR-MB患者。A组（2016 - 2019年）接受了儿童肿瘤学组（COG）A9961方案A的辅助化疗，数据为回顾性收集。B组（2020年 - 2022年7月）接受了ACNS0331方案的化疗，数据为前瞻性收集。使用不良事件通用术语标准（CTCAE）v5.0评估和分级毒性。
共纳入168例3至18岁患者，A组112例（67%），B组56例（33%）。A组≥3级耳毒性显著更高（24%对3.6%，p < 0.001）。神经毒性发生率分别为26%对12.5%（p = 0.046）。贫血和血小板减少发生率分别为71%对52%（p = 0.04）。B组发热性中性粒细胞减少更常见（66%对38%，p < 0.001）。≥3级白细胞减少、肾毒性或肝毒性无显著差异。A组2年总生存率为96.4%（95%CI：93.1 - 99.9），B组为86.6%（95%CI：77.8 - 96.）（p =）。无事件生存率分别为92.9%（95%CI：88.2 - 97.8）对86.8%（95%CI：78 - 96.4）（p = 0.29）。
用环磷酰胺部分替代顺铂显示出更好的毒性特征，尤其是耳毒性和神经毒性，生存率无显著差异。
