Nguyen Peter, Indelicato Daniel J, Esterman Adrian, Paulino Arnold C, Ermoian Ralph P, Laack Nadia N, Perkins Stephanie M, Mangona Victor, Mihalcik Stephen, Lee Jae, Hill-Kayser Christine, Kwok Young, Chang John Han-Chih, Perentesis John P, MacEwan Iain, Le Hien, Yock Torunn I
Royal Adelaide Hospital, Adelaide, Australia.
University of Florida Health Proton Therapy Institute, Jacksonville, Florida, USA.
Int J Part Ther. 2025 Apr 17;16:100747. doi: 10.1016/j.ijpt.2025.100747. eCollection 2025 Jun.
Medulloblastoma is one of the most common malignant brain tumors in children, and 75% of patients achieve long-term survival. There are limited studies reporting acute toxicity for pediatric patients receiving proton therapy for craniospinal irradiation (CSI) in medulloblastoma, and these are limited by their retrospective nature, modest cohort sizes, and lack of a comparator group.
We analyzed data from the Pediatric Proton/Photon Consortium Registry. Patients with a diagnosis of medulloblastoma, receiving CSI with doses of either 23.4 Gy or 36 to 39.6 Gy, and with toxicity data at baseline and completion of treatment, were identified for inclusion.
A total of 272 patients were included for analysis. All patients received proton therapy. The median age of patients was 8 years (range 3-22 years), and 67.6% were male. Most patients were of good performance status with eastern cooperative oncology group (ECOG) 0 or 1, 36.8% and 31.6%, respectively. In total, 68.8% of patients had classic medulloblastoma; 76.8% had M0 disease; and 62.9% of patients had standard-risk disease.Acute toxicities were reported as grade 1 or higher. The most common toxicities occurring during treatment were skin (90.3%), gastrointestinal (71.6%), hematological (54.9%), and mouth (32.0%). Toxicity rates were lower than in the published literature. All patients completed treatment.
Our findings suggest that proton CSI has an acceptable degree of acute toxicity in the treatment of pediatric medulloblastoma. Future studies would benefit from toxicity grading, toxicity timing, and a photon comparator group.
髓母细胞瘤是儿童最常见的恶性脑肿瘤之一，75%的患者可实现长期生存。关于接受质子治疗进行髓母细胞瘤全脑全脊髓照射（CSI）的儿科患者的急性毒性的研究有限，且这些研究受限于其回顾性性质、样本量较小以及缺乏对照组。
我们分析了儿科质子/光子联合登记处的数据。纳入诊断为髓母细胞瘤、接受23.4 Gy或36至39.6 Gy剂量CSI且有基线和治疗结束时毒性数据的患者。
共纳入272例患者进行分析。所有患者均接受质子治疗。患者的中位年龄为8岁（范围3 - 22岁），67.6%为男性。大多数患者的东部肿瘤协作组（ECOG）体能状态良好，分别为0或1级，比例分别为36.8%和31.6%。总体而言，68.8%的患者患有经典型髓母细胞瘤；76.8%为M0期疾病；62.9%的患者为标准风险疾病。报告的急性毒性为1级或更高。治疗期间最常见的毒性是皮肤（90.3%）、胃肠道（71.6%）、血液学（54.9%）和口腔（32.0%）。毒性发生率低于已发表的文献。所有患者均完成了治疗。
我们的研究结果表明，质子CSI在儿科髓母细胞瘤治疗中的急性毒性程度可接受。未来的研究将受益于毒性分级、毒性发生时间以及光子治疗对照组。
