Molecular and clinical correlates of medulloblastoma subgroups: A narrative review

Abstract

Medulloblastoma is a major cause of cancer-related morbidity and mortality in children, as a significant proportion of patients succumb to their disease and most survivors are left with life-long sequelae of therapy. Prior medulloblastoma classification systems relied heavily on histology and failed to account for tumor biology. The upcoming 2021 WHO classification of central nervous system tumors now firmly establishes that medulloblastoma actually comprises at least four distinct molecular entities, with considerable substructure within each group. For the first time, the study design of contemporary clinical trials has now recognized the molecular heterogeneity of medulloblastoma. The incorporation of routine molecular subgrouping into upcoming clinical trials has the potential to significantly improve survival and quality of life for children and adults diagnosed with medulloblastoma. This review was conducted to summarize these recent advances in the genomics of medulloblastoma and to summarize the timely results of molecularly-informed published clinical trials. Specifically, English language literature will be reviewed in addition to the results of SJMB03, ACNS0331, and ACNS0332.