What Have We Learnt from the Recent Multimodal Managements of Young Patients with ATRT?

Atypical teratoid rhabdoid tumors are rare embryonal tumors of the nervous system mainly seen in very young children with aggressive behavior and dismal prognosis when treated with conventional chemotherapy only. More recent multimodal strategies combining, variably, high dose chemotherapy, radiotherapy and or intrathecal chemotherapy have led to some stride in survival. We present the results of the most recent clinical trials and registry data for patients treated with these multimodal approaches with survival ranging from 37.1% to 88.9%. We review the current consensus of the molecular characterization of these tumors into 3 subgroups (ATRT-TYR, ATRT-SHH and ATRT-MYC) and discuss the potential clinical impact of molecular subgrouping on survival. We explore other therapeutic tools including intrathecal chemotherapy and maintenance and possible new targeted agents for patients failing multimodal strategies.