Radiation for ETMR: Literature review and case series of patients treated with proton therapy

Background and purpose: Embryonal tumors with multilayered rosettes (ETMRs) are aggressive tumors that typically occur in young children. Radiation is often deferred or delayed for these patients due to late effects; proton therapy may mitigate some of these concerns. This study reviews the role of radiation in ETMR and describes initial results with proton therapy. Materials and methods: Records of patients with embryonal tumor with abundant neuropil and true rosettes (ETANTR), medulloepithelioma (MEP), and ependymoblastoma (EPL) treated with proton therapy at our institution were retrospectively reviewed. A literature review of cases of CNS ETANTR, MEP, and EPL published since 1990 was also conducted. Results: Seven patients were treated with proton therapy. Their median age at diagnosis was 33 months (range 10–57 months) and their median age at radiation start was 42 months (range 17–58 months). Their median overall survival (OS) was 16 months (range 8–64 months), with three patients surviving 36 months or longer. Five patients had disease progression prior to starting radiation; all 5 of these patients failed in the tumor bed. A search of the literature identified 204 cases of ETMR with a median OS of 10 months (range 0.03–161 months). Median OS of 18 long-term survivors (≥36 months) in the literature was 77 months (range 37–184 months). Of these 18 long-term survivors, 17 (94%) received radiotherapy as part of their initial treatment; 14 of them were treated with craniospinal irradiation. Conclusions: Outcomes of patients with ETMR treated with proton therapy are encouraging compared to historical results. Further study of this rare tumor is warranted to better define the role of radiotherapy.