Current treatment approaches to early childhood medulloblastoma.

Treatment of medulloblastoma, the most common malignant brain tumor of childhood, is particularly challenging in very young children, owing to the increased susceptibility of the immature brain to treatment-induced neurocognitive deficits. Three promising strategies have been developed in combination with systemic postoperative chemotherapy, to avoid craniospinal irradiation for young children with nonmetastatic medulloblastoma, these include: high-dose chemotherapy, with and without local radiotherapy; intraventricular chemotherapy; and local radiotherapy. More intensified strategies may be required for metastatic medulloblastoma. Future studies will clarify the prognostic relevance of desmoplasia, postoperative residual tumor and biological markers to improve stratification criteria by risk-adapted treatment recommendations. An international Phase III trial for young children with nonmetastatic medulloblastoma, comparing survival rates and neurocognitive outcomes of different treatment strategies by standardized criteria, is under discussion.