Treatment of children aged 3 years and younger

The optimal treatment of childhood nonmedulloblastoma embryonal tumors remains unclear and under study. Retrospective studies of fairly large numbers of patients have suggested management approaches for the more common subgroups, including AT/RTs, ETMRs, and FOXR2-activated tumors. For more information about the treatment of AT/RTs, see Childhood Central Nervous System Atypical Teratoid/Rhabdoid Tumor Treatment.

Standard treatment options for children aged 3 years and younger with newly diagnosed nonmedulloblastoma embryonal tumors, excluding AT/RTs, ETMRs, and FOXR2-activated tumors, include the following:

Treatment of children aged 3 years and younger with embryonal tumors is similar to that outlined for children aged 3 years and younger with medulloblastoma. Aggressive surgical resection is reasonable, given the improved rate of survival for medulloblastomas and other ETMRs after total or near-total resection.[11] For more information, see the Treatment of younger children with medulloblastoma section.

With the use of chemotherapy alone, outcome has been variable, with survival rates at 5 years ranging between 0% and 50%.[24-26]; [27][Level of evidence B4] The addition of craniospinal irradiation to chemotherapy-based regimens may successfully treat some children but with anticipated neurodevelopmental decline.[28][Level of evidence B4] Localized radiation therapy to the tumor site, either before or after chemotherapy, has been given, although data supporting its efficacy are unclear.