Diaz-Coronado Rosdali Y, Alcasabas Ana Patricia A, Baticulon Ronnie E, Hamid Syed Ahmer, Omran Nuha, Sharafeldin Hend, Pineda Estuardo, Navarro-Martín Del Campo Regina M, Foo Jen Chun, Ganesan Dharmendra, Rajagopal Revathi, Escamilla Gabriela, Amarillo Paloma, Villegas César A, Job Godwin, Campos Danny, Hernandez-Broncano Eddy, Syed Yumna, Yousuf Bisma, Fadel Shady H, Seddik Khaled, Polanco Ana, Arredondo Luis, Garcia Beatriz, Devidas Meenakshi, Qaddoumi Ibrahim, Moreira Daniel C
Department of Pediatric Oncology, Instituto Nacional de Enfermedades Neoplásicas, Lima, Peru.
Philippine General Hospital, University of the Philippines Manila, Manila, Philippines.
Neurooncol Adv. 2025 Apr 24;7(1):vdaf086. doi: 10.1093/noajnl/vdaf086. eCollection 2025 Jan-Dec.
Reports from low- and middle-income countries (LMICs) indicate lower survival for children with medulloblastoma (MB), though these studies are mainly from single institutions or countries. This retrospective study aimed to analyze the outcomes for MB in 9 centers in 8 countries.
Patients <18 years with newly diagnosed MB, diagnosed between 2014 and 2018, were included. Clinical and biological characteristics were collected, and patient outcomes were analyzed.
A total of 335 patients were included. The median age was 7.7 years, and 51 patients (15%) were <3 years old. Eighty-seven patients (26.0%) had experienced symptoms for more than 3 months before diagnosis. Of 326 (97.3%) patients who underwent resection, 153 (47%) had a gross total resection. Metastatic disease at diagnosis was identified in 82 patients (24.5%). Radiotherapy was administered to 213 patients (63.8%). A total of 117 deaths occurred, including 90 (76.9%) from tumor progression and 25 (21.4%) from surgical complications or infection. Sixty-three (18.9%) patients abandoned treatment. The 5-year event-free survival and overall survival (OS) were 43.8% ± 3.5% and 58.4% ± 3.7%, respectively. Younger age, presence of metastatic disease, and starting radiotherapy >42 days after diagnosis were not prognostic, while the degree of resection, presence of a severe infection, and a longer diagnostic interval were associated with worse outcomes.
This multinational cohort describes poor outcomes due to delays in care, high abandonment of treatment, surgical complications, and a low fraction of patients receiving radiotherapy. Interventions to build care capacity for children with MB in LMICs can be prioritized based on these data.
来自低收入和中等收入国家（LMICs）的报告显示，髓母细胞瘤（MB）患儿的生存率较低，不过这些研究主要来自单一机构或国家。这项回顾性研究旨在分析8个国家9个中心的MB治疗结果。
纳入2014年至2018年间新诊断为MB的18岁以下患者。收集临床和生物学特征，并分析患者的治疗结果。
共纳入335例患者。中位年龄为7.7岁，51例（15%）患者年龄小于3岁。87例（26.0%）患者在诊断前出现症状超过3个月。在326例（97.3%）接受手术切除的患者中，153例（47%）实现了全切除。82例（24.5%）患者在诊断时发现有转移性疾病。213例（63.8%）患者接受了放疗。共发生117例死亡，其中90例（76.9%）死于肿瘤进展，25例（21.4%）死于手术并发症或感染。63例（18.9%）患者放弃治疗。5年无事件生存率和总生存率（OS）分别为43.8%±3.5%和58.4%±3.7%。年龄较小、存在转移性疾病以及诊断后>42天开始放疗并非预后因素，而切除程度、严重感染的存在以及较长的诊断间隔与较差的治疗结果相关。
这个跨国队列描述了由于护理延迟、高放弃治疗率、手术并发症以及接受放疗的患者比例低而导致的不良治疗结果。基于这些数据，可以优先考虑在低收入和中等收入国家为患有MB的儿童建立护理能力的干预措施。
