Abstract
Simple SummaryMedulloblastoma 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.
Highlights
Medulloblastoma (MB) is the most common malignant brain tumor in childhood
The WHO consensus conference held in June 2015 recognized the importance of these biological groups, and the revised WHO classification of central nervous system (CNS) tumors published in 2016 defined MB both histologically and genetically [4]
We present the most recent trials using high-dose chemotherapy (HDC) regimens for HR and/or young patients with MB, and we discuss their contribution to improved survival and describe their toxicity profile and limitations
Summary
Medulloblastoma (MB) is the most common malignant brain tumor in childhood. Based on gene expression patterns in tumor tissue, MB can be classified into distinct subgroups. Strother et al, investigated the feasibility of intensive chemotherapy regimens after surgical resection and CSI in the front-line treatment of children with MB and primitive neuro-ectodermal tumors (PNET) [10] In this pilot study, after surgical resection, 19 patients with HR MB were treated with Topotecan in a 6-week phase II window, followed by risk-adapted CSI (M0-M1, 36 Gy; M2/M3, 39.6 Gy) and three-dimensional conformal boost to tumor bed (total dose, 55.8 Gy) and, where appropriate, local sites of metastases (total dose, 50.5 Gy). Fifty-one patients with HR MB were treated with 2 courses of conventional chemotherapy (Etoposide, Carboplatin), 2 courses of high-dose Thiotepa (200 mg/m2/day for 3 days), followed by conventional CSI (36 Gy on neuraxis; 54 Gy on primitive tumor bed) and maintenance treatment based on Temozolomide. Gy: Gray; CPM: cyclophosphamide; CDDP: cisplatin; VCR: vincristine; MTX: methotrexate; CBP: carboplatin; TMZ: temozolomide; CR: complete remission
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