Pediatric embryonal brain tumors in the molecular era
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Bryan K Li1,2,3, Salma Al-Karmi2, Annie Huang1,2,3
1Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, ON, Canada.
Insights
This review covers embryonal brain tumors (EBTs), including medulloblastoma and others. Advances in molecular classification offer new insights but require further research for improved high-risk patient survival.
Area of Science:
- Pediatric Oncology
- Neuro-oncology
- Cancer Genomics
Background:
- Embryonal brain tumors (EBTs) are aggressive pediatric malignancies.
- Includes medulloblastoma, ATRT, pineoblastoma, ETMR, and FOXR2/BCOR-altered tumors.
- Diagnosis is evolving from histopathology to molecular classification.
Purpose of the Study:
- Provide a comprehensive literature overview of EBTs.
- Discuss the shift towards molecular-based classification.
- Review current and emerging treatment modalities.
Main Methods:
- Literature review of embryonal brain tumors.
- Analysis of diagnostic evolution and molecular subtyping.
- Evaluation of treatment strategies and clinical trial implications.
Main Results:
- Molecular subgrouping enhances risk stratification and identifies driver pathways.
- Multi-omic profiling is crucial for understanding EBT heterogeneity.
- Current molecular insights guide targeted therapy development.
Conclusions:
- Molecular classification represents a significant advancement in EBT understanding.
- Further preclinical and clinical validation is needed to improve outcomes for high-risk patients.
- Integration of multi-omic data into clinical trials is essential for therapeutic progress.
Abstract:
Introduction: Embryonal brain tumors (EBTs) are highly aggressive malignancies predominantly affecting children. They include medulloblastoma (MB), atypical rhabdoid/teratoid tumors (ATRT), pineoblastoma (PB), embryonal tumor multiple rosettes (ETMR)/C19MC-altered tumors, and newly recognized embryonal tumors with FOXR2 activation or BCOR alteration.Areas covered: This review will provide a comprehensive overview and updated of the literature on each of these EBTs. The evolution from location- and histopathology-based diagnosis to more specific and robust molecular-based classification schemes, as well as treatment modalities, will be discussed.Expert commentary: The subgrouping of EBTs with multi-omic profiling has had important implications for risk stratification and discovery of targetable driver pathways. However, these innovations are unlikely to significantly improve survival among high-risk patients until robust preclinical studies are conducted, followed by validation in biology-informed clinical trials.
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