J Korean Med Assoc JKMA Journal of the Korean Medical Association 1975-8456 2093-5951 Korean Medical Association 10.5124/jkma.2012.55.5.430 Focused Issue of This Month Pediatric Brain Tumors High-dose chemotherapy and autologous stem cell transplantation for pediatric brain tumors Lee Soo Hyun MD Sung Ki Woong MD Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea. Corresponding author: Ki Woong Sung, kwsped@skku.edu 05 2012 16 05 2012 55 5 430 437 28 03 2012 11 04 2012 Copyright © 2012 Korean Medical Association 2012

This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/3.0) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.

The prognosis of brain tumors in children has improved for the last 2-3 decades. However, the prognosis remains dismal in patients with relapsed tumors. The outcome for infants and young children is also poor. For younger children, the ability to use of radiotherapy (RT) is very limited because of the unacceptable long-term adverse effects of RT. The prognosis is also not satisfactory when a large residual tumor remains after surgery or when leptomeningeal seeding is present at diagnosis. In this context, a strategy using high-dose chemotherapy and autologous stem cell transplantation (HDCT/autoSCT) has been explored to improve the prognosis of recurrent or high-risk brain tumors. It was found that at least some patients with relapsed tumors can be salvaged with HDCT/autoSCT. For infants and young children, it was possible to avoid or defer RT until 3 years of age while maintaining or improving survival rates. Investigators also have explored the efficacy of HDCT/autoSCT in patients with newly diagnosed embryonal tumors to further improve the survival rate or to reduce the craniospinal RT dose without jeopardizing the survival rate. Preliminary results were encouraging although the numbers of patients was small. Recently, a few investigators have evaluated the efficacy of sequential HDCT/autoSCT to further improve the outcome. This strategy is based on the hypothesis that further dose escalation might result in further improvement in survival rates. At present, the number of studies employing a sequential HDCT/autoSCT strategy is limited. However, preliminary results of these studies suggest that sequential HDCT/autoSCT may further improve outcomes.

 Brain neoplasms Child High-dose chemotherapy Autologous stem cell transplantation Acknowledgement

This study was supported by a grant from the National R&D Program for Cancer Control, Ministry of Health and Welfare, Republic of Korea (no. 0520300).

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HDCT/autoSCT in recurrent brain tumors

HDCT, high-dose chemotherapy; autoSCT, autologous stem cell transplantation; CCG, Children's Cancer Group; MB, medulloblastoma; CTE, carboplatin+thiotepa+etoposide; EFS, event-free survival; BuT, busulfan+thiotepa; RT, radiotherapy; OS, overall survival; BT, brain tumor; PFS, progression-free survival; PNET, primitive neuroectodermal tumor; CT, chemotherapy; CyM, cyclophosphamide+melphalan; KSPNO, Korean Society of Pediatric Neuro-Oncology.

HDCT/autoSCT in young children with malignant brain tumors

HDCT, high-dose chemotherapy; autoSCT, autologous stem cell transplantation; RT, radiotherapy; BT, brain tumor; CT, chemotherapy; CTE, carboplatin+thiotepa+etoposide; R+, residual tumor; EFS, event-free survival; OS, overall survival; MB, medulloblastoma; HD-MTX, high-dose methotrexate; CSI, craniospinal irradiation; CaT, carboplatin+thiotepa; M+, leptomeningeal seeding; PFS, progression-free survival; PNET, primitive neuroectodermal tumor; CyM, cyclophosphamide+melphalan; R0, no residual tumor; M0, no leptomeningeal seeding; BuM, busulfan+melphalan; KSPNO, Korean Society of Pediatric Neuro-Oncology; AE, anaplastic ependymoma; ATRT, atypical teratoid/rhabdoid tumor.

HDCT/autoSCT in newly diagnosed high-risk embryonal tumors

HDCT, high-dose chemotherapy; autoSCT, autologous stem cell transplantation; RT, radiotherapy; MB, medulloblastoma; AR, average-risk; HR, high-risk; CSI, craniospinal irradiation; CDDP, cisplatin; Cy, cyclophosphamide; VCR, vincristine; EFS, event-free survival; OS, overall survival; PNET, primitive neuroectodermal tumor; CyM, Cy+melphalan; CTE, carboplatin+thiotepa+etoposide.

HDCT/autoSCT in high-grade glioma

HDCT, high-dose chemotherapy; autoSCT, autologous stem cell transplantation; RT, radiotherapy; GM, glioblastoma multiforme; TE, thiotepa+etoposide; BTE, BCNU (carmustine)+TE; CTE, carboplatin+TE; EFS, event-free survival; AA, anaplastic astrocytoma; CCG, Children's Cancer Group; PFS, progression-free survival; AO, anaplastic oligodendroglioma; CT, chemotherapy; T, thiotepa.