Abstract
Simple SummaryBrain tumors remain the most common childhood solid tumors, accounting for approximately 25% of all pediatric cancers. They also represent the most common cause of cancer-related illness and death in this age group. Recent years have witnessed an evolution in our understanding of the biological underpinnings of many childhood brain tumors, potentially improving survival through both improved risk group allocation for patients to provide appropriate treatment intensity, and novel therapeutic breakthroughs. This review aims to summarize the molecular landscape, current trial-based standards of care, novel treatments being explored and future challenges for the three most common childhood malignant brain tumors—medulloblastomas, high-grade gliomas and ependymomas.Brain tumors are the leading cause of childhood cancer deaths in developed countries. They also represent the most common solid tumor in this age group, accounting for approximately one-quarter of all pediatric cancers. Developments in neuro-imaging, neurosurgical techniques, adjuvant therapy and supportive care have improved survival rates for certain tumors, allowing a future focus on optimizing cure, whilst minimizing long-term adverse effects. Recent times have witnessed a rapid evolution in the molecular characterization of several of the common pediatric brain tumors, allowing unique clinical and biological patient subgroups to be identified. However, a resulting paradigm shift in both translational therapy and subsequent survival for many of these tumors remains elusive, while recurrence remains a great clinical challenge. This review will provide an insight into the key molecular developments and global co-operative trial results for the most common malignant pediatric brain tumors (medulloblastoma, high-grade gliomas and ependymoma), highlighting potential future directions for management, including novel therapeutic options, and critical challenges that remain unsolved.
Highlights
Brain tumors are the most common solid tumors of childhood, accounting for approximately 25% of all pediatric malignancies, and represent the leading cause of cancer-induced morbidity and mortality in this age group [1]
Medulloblastoma (MB) represents the most common malignant brain tumor in children, accounting for approximately 20% of all central nervous system (CNS) tumors [2,4]. It comprises over 60% of intracranial embryonal tumors, a recently characterized entity consisting of atypical teratoid rhabdoid tumors (ATRTs), embryonal tumors with multilayer rosettes (ETMRs), CNS neuroblastoma with FOX2 alteration and malignant neuroepithelial tumors with BCOR alteration [5]
Despite a paradigm shift in our understanding of pediatric high-grade gliomas (pHGGs) molecular subgrouping being distinct from adult counterparts, and some therapeutic successes for particular entities, little progress has been made over recent decades to improve the dismal prognosis; pHGGs account for over 40% of all childhood brain tumor deaths [81]
Summary
Brain tumors are the most common solid tumors of childhood, accounting for approximately 25% of all pediatric malignancies, and represent the leading cause of cancer-induced morbidity and mortality in this age group [1]. For the majority of brain tumors, prognosis has remained static for over 30 years despite these technological improvements To overcome this impasse, the pediatric neuro-oncology community has shifted focus to develop risk-stratified treatment protocols that aim to reduce iatrogenic morbidity while maintaining outcomes for favorable-risk lesions, and improve cure rates for tumors refractory to conventional therapy, either through intensification or novel agents. The pediatric neuro-oncology community has shifted focus to develop risk-stratified treatment protocols that aim to reduce iatrogenic morbidity while maintaining outcomes for favorable-risk lesions, and improve cure rates for tumors refractory to conventional therapy, either through intensification or novel agents This strategy has been supplemented by an evolution in our understanding of the molecular pathogenesis of almost all pediatric brain tumors. This article will provide a summary of the most common malignant pediatric brain tumors (medulloblastoma, high-grade gliomas and ependymoma) with particular focus on inherent molecular advancements and potential future directions for management, including novel therapeutic options
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