ORIGINAL RESEARCH article
Front. Oncol.
Sec. Pediatric Oncology
Volume 15 - 2025 | doi: 10.3389/fonc.2025.1597123
Molecular Subtypes, Prognostic Factors, and Treatment Optimization in Pediatric Medulloblastoma: A Real-World Study from China
Provisionally accepted
- 1Beijing Shijitan Hospital, Capital Medical University, Beijing, China
- 2Department of Pulmonary and Critical Care Medicine, Sixth Medical Center of PLA General Hospital, Beijing, China
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Background: Medulloblastoma (MB) is the most common malignant pediatric brain tumor, yet systematic studies on molecular characteristics and treatment efficacy in Chinese pediatric MB remain scarce. This study evaluates recurrence and mortality risk factors and the feasibility of intensified chemotherapy.Methods: A retrospective analysis of 381 MB patients (WNT: 63, SHH: 106, Group 3: 27, Group 4: 185) was conducted. Kaplan-Meier analysis estimated survival rates, and Cox regression identified independent risk factors for recurrence and mortality.Results: With a median follow-up of 4.8 years, 5-year PFS and OS were 69.9%±2.4% and 80.6%±2.1%, respectively. WNT-MB had the best prognosis, while Group 3-MB had the worst.Independent recurrence risk factors included high-risk status (HR=2.931, p<0.001), TP53 mutation (HR=1.873, p<0.001), MYCN amplification (HR=1.52, p=0.001), chromosome 12p amplification, and 9q deletion. Mortality was associated with LC/A pathology (HR=1.573, p=0.007), TP53 mutation (HR=2.049, p<0.001), and high-risk status (HR=3.966, p<0.001). TP53 mutations influenced WNT-MB recurrence, and Group 3-MB showed a high recurrence risk even without MYC amplification or metastasis. No treatment-related fatalities were observed.This study identified key clinical and molecular risk factors associated with recurrence and mortality in pediatric medulloblastoma. The findings underscore the prognostic relevance of TP53 mutations, MYCN amplification, and specific chromosomal alterations, particularly in non-metastatic subgroups. These insights may help guide risk-adapted and personalized treatment strategies in future studies.
Keywords: Medulloblastoma, risk factors for relapse and mortality, maintain chemotherapy by risk stratification, Large cohort, Real-world study
Received: 20 Mar 2025; Accepted: 08 Jul 2025.
Copyright: © 2025 Liu, Sun, Li, Shuxu, Zhang, Zhang, Sun and Wu. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Wanshui Wu, Beijing Shijitan Hospital, Capital Medical University, Beijing, China
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