Abstract

Despite recent progress in molecular diagnostics defining four distinct medulloblastoma groups, the clinical management of these malignant childhood tumors of the cerebellum remains challenging. After surgical removal of the tumor, both cytotoxic chemotherapy and irradiation can offer additional curative benefits, but they also include a significant risk of long-term damage. Early molecular profiling aims to predict the outcome of such aggressive therapies. This prevents unnecessary damage to patients who may not need it and helps to identify those patients with remaining tumor cells who may benefit from more aggressive treatment with the intent to cure. Monitoring tumor evolution in real time allows personalized precision medicine with an immediate clinical response resulting in a better outcome. Liquid biopsy includes various methodologies already applied in numerous studies and clinical trials for common cancers including brain tumors, but information on medulloblastomas is limited. This review summarizes the recent developments of how liquid biopsy can support or even replace the standard monitoring of medulloblastomas by medical imaging or cytology and discusses what will be needed to make liquid biopsy a new gold standard in diagnosis, therapy, and follow-up of medulloblastomas for the benefit of the patients.

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