High-Dose Chemotherapy in Children with Newly Diagnosed Medulloblastoma

Simple Summary Medulloblastoma is the most common malignant central nervous system tumor in the pediatric population. Treatment modalities are stratified by age, extent of resection, metastatic status, histology, and more recently, tumor biology. Maximal surgical resection followed by risk-adapted craniospinal irradiation and adjuvant chemotherapy have produced the best survival for medulloblastoma. Although survival is, in general, quite high, some caveats and drawbacks argue for continuous adaptations of this treatment strategy. Clearly, for children with metastatic disease, there is no established standard chemotherapy regimen, even if there is a consensus that chemotherapy brings additional benefit. The treatment of infants and very young children is particularly challenging, as irradiation of the developing brain leads to substantial neurocognitive impairment. Some clinical trials have explored the possible efficacy of high-dose chemotherapy in childhood medulloblastoma, and here we have focused on clinical trials for infants and children with newly diagnosed medulloblastoma. High-dose chemotherapy with stem cell rescue has been used as an adjuvant therapy or as salvage therapy to treat pediatric patients with brain tumors, and to avoid deleterious side effects of radiotherapy in infants and very young children. Here, we present the most recent trials using high-dose chemotherapy regimens for medulloblastoma in children, and we discuss their contribution to improved survival and describe their toxicity profile and limitations.