Childhood medulloblastoma: current and future treatment strategies

Abstract

Introduction: Medulloblastoma is the most common malignant brain tumor in children. Standard of care treatment for children ≥ 3 years has resulted in a cure rate of approximately 70 – 75%. However, treatment-related toxicity often has a major impact on long-term quality of survival.

Areas covered: This article summarizes the epidemiology, clinical presentation, radiologic features, pathology, prognosis, risk stratification, molecular genetics and current and future treatment strategies of medulloblastoma.

Expert opinion: Treatment of children with newly diagnosed medulloblastoma includes maximal surgical resection, risk-adapted craniospinal radiotherapy plus a focal boost to the tumor bed, and dose-intensive chemotherapy consisting of cisplatin, vincristine and cyclophosphamide and lomustine. Recent studies using next-generation sequencing have identified the complex molecular heterogeneity of this malignant tumor allowing a more precise identification of prognostic factors and specific oncogenic targets for tumor control. Future patients will be enrolled in clinical trial(s) that will identify the molecular subgroups in real time, allowing for de-escalation of therapy for patients with low- or average-risk disease and treatment intensification for those with high-risk tumors. Ongoing laboratory research is striving to identify novel agents that target specific driver mutations in each molecular subgroup for those patients with recurrent or refractory disease.

Keywords:

• brain tumor
• childhood
• medulloblastoma
• molecular subgroups
• pediatric
• targeted
• therapy
• treatment

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending or royalties.

Notes

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