Guerra JA, et al. – This study aspired to probe irradiation–avoiding or –minimizing marrow–ablative chemotherapy (HDCx) with autologous hematopoietic cell transplantation (AuHCT), in order to improve survival in young children with malignant brain tumors. For this group of patients, HDCx with tandem AuHCT was feasible and safe.

• The outcome of 44 children with malignant brain tumors treated with HDCx and tandem AuHCT at Children’s Hospital Los Angeles between June 1999 and July 2012, were assessed.
• Experts examined forty-four children with malignant brain tumors.
• In addition, twenty-one had medulloblastoma/primitive neuro-ectodermal tumor, eight atypical teratoid/rhabdoid tumor (ATRT), five high-grade glioma, four malignant germ cell tumor, three ependymoma and three choroid plexus carcinoma.
• Three tandem transplants were given to twenty-nine patients, and 15 received two tandem transplants, respectively.
• 46.3±8.2% and 51.7±8.5% were the 5-year PFS and overall survivals (OS) for all patients, respectively.
• As compared with 17 transplanted at relapse 11.8±9.8% (P<0.001) and 15.1±12.3%(P=0.0231), the PFS and OS for 27 newly diagnosed patients were 68.9±9.9% and 73.5±9.3%.
• It was found that the 5-year PFS and OS in 13 previously unirradiated patients were 74±13% and 74±13% versus 33.2±9.8%and 40.2±10.6% in 31 irradiated patients (P=0.11 and P=0.239), respectively.
• Transplant-related toxicity was the cause of death in one patient