{"title":"Advancing medulloblastoma therapy: strategies and survival insights.","authors":"Zhenjiang Pan, Jing Bao, Shepeng Wei","doi":"10.1007/s10238-025-01648-5","DOIUrl":null,"url":null,"abstract":"<p><p>Medulloblastoma, the most common malignant brain tumor in children, presents unique challenges due to its molecular and histological heterogeneity. Advances in molecular profiling have refined risk stratification, enabling personalized treatment strategies and improved survival outcomes. This review synthesizes recent developments in the multimodal management of medulloblastoma, encompassing surgery, craniospinal radiation therapy, and chemotherapy, tailored to patient age and risk classification. Key highlights include subgroup-specific therapies, the role of molecular-targeted treatments, and the integration of genetic testing for germline mutations to guide clinical decision-making. Special emphasis is placed on minimizing treatment-related toxicity while preserving long-term quality of life. Additionally, this manuscript discusses the implications of novel therapeutic approaches for high-risk subgroups, including intensified regimens and systemic therapies for young children. Despite significant progress, challenges remain in addressing long-term complications such as neurocognitive impairments, endocrine dysfunction, and secondary malignancies. Future directions prioritize optimizing therapeutic efficacy while reducing morbidity, underscoring the importance of translating molecular discoveries into clinical practice.</p>","PeriodicalId":10337,"journal":{"name":"Clinical and Experimental Medicine","volume":"25 1","pages":"119"},"PeriodicalIF":3.2000,"publicationDate":"2025-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12003599/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Clinical and Experimental Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s10238-025-01648-5","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}
引用次数: 0
Abstract
Medulloblastoma, the most common malignant brain tumor in children, presents unique challenges due to its molecular and histological heterogeneity. Advances in molecular profiling have refined risk stratification, enabling personalized treatment strategies and improved survival outcomes. This review synthesizes recent developments in the multimodal management of medulloblastoma, encompassing surgery, craniospinal radiation therapy, and chemotherapy, tailored to patient age and risk classification. Key highlights include subgroup-specific therapies, the role of molecular-targeted treatments, and the integration of genetic testing for germline mutations to guide clinical decision-making. Special emphasis is placed on minimizing treatment-related toxicity while preserving long-term quality of life. Additionally, this manuscript discusses the implications of novel therapeutic approaches for high-risk subgroups, including intensified regimens and systemic therapies for young children. Despite significant progress, challenges remain in addressing long-term complications such as neurocognitive impairments, endocrine dysfunction, and secondary malignancies. Future directions prioritize optimizing therapeutic efficacy while reducing morbidity, underscoring the importance of translating molecular discoveries into clinical practice.
期刊介绍:
Clinical and Experimental Medicine (CEM) is a multidisciplinary journal that aims to be a forum of scientific excellence and information exchange in relation to the basic and clinical features of the following fields: hematology, onco-hematology, oncology, virology, immunology, and rheumatology. The journal publishes reviews and editorials, experimental and preclinical studies, translational research, prospectively designed clinical trials, and epidemiological studies. Papers containing new clinical or experimental data that are likely to contribute to changes in clinical practice or the way in which a disease is thought about will be given priority due to their immediate importance. Case reports will be accepted on an exceptional basis only, and their submission is discouraged. The major criteria for publication are clarity, scientific soundness, and advances in knowledge. In compliance with the overwhelmingly prevailing request by the international scientific community, and with respect for eco-compatibility issues, CEM is now published exclusively online.