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.