High-dose chemotherapy and autologous stem cell rescue in children with newly diagnosed high-risk or relapsed medulloblastoma or supratentorial primitive neuroectodermal tumor.
BACKGROUND
Single or tandem double high-dose chemotherapy (HDCT) was used to treat children with newly diagnosed high-risk or relapsed medulloblastoma and supratentorial primitive neuroectodermal tumor (MB/sPNET) in order to defer or avoid radiotherapy in young children.


METHODS
Thirty-seven HDCTs were given to 25 children with newly diagnosed high-risk or relapsed MB/sPNET. Tandem double HDCT was used for 12 of 15 patients initially intended to receive double HDCT.


RESULTS
Three-year EFS (+/-SE) in 6 newly diagnosed high-risk (>3 years old), 8 newly diagnosed (<3 years old), and 11 relapsed MB/sPNET was 83.3 +/- 15.2%, 62.5 +/- 20.5%, and 29.1 +/- 15.7%, respectively. Three-year EFS for patients in CR or PR and in less than PR at first HDCT was 67.4 +/- 11.0% and 16.7 +/- 15.2%, respectively (P = 0.001). Three-year EFS in patients initially intended to receive double HDCT and single HDCT was 66.0 +/- 12.4% and 40.0 +/- 15.5%, respectively. For 19 patients in CR or PR at first HDCT, 3-year EFS was 88.9 +/- 10.5% in tandem double HDCT group, and 44.4 +/- 16.6% in single HDCT group, respectively (P = 0.037). Although four treatment-related mortalities (TRMs) occurred during 25 first HDCTs, no TRM occurred during 12 second HDCTs. In four of eight young children, craniospinal radiotherapy was successfully withheld without subsequent relapse.


CONCLUSIONS
High-dose chemotherapy may improve the survival of children with newly diagnosed high-risk MB/sPNET, and, to some extent, the survival of those with relapsed MB/sPNET. Further study is necessary to elucidate the efficacy of tandem double HDCT.
Sung
Ki Woong
KW
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
Yoo
Keon Hee
KH
Cho
Eun Joo
EJ
Koo
Hong Hoe
HH
Lim
Do Hoon
DH
Shin
Hyung Jin
HJ
Ahn
Seung Do
SD
Ra
Young Shin
YS
Choi
Eun Seok
ES
Ghim
Thad T
TT
D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D002528:Cerebellar Neoplasms / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000601:surgery / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D002945:Cisplatin / Q000008:administration & dosage / Q000009:adverse effects; D000075202:Contraindications; D016371:Cranial Irradiation; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000009:adverse effects; D005047:Etoposide / Q000008:administration & dosage / Q000009:adverse effects; D005260:Female; D005500:Follow-Up Studies; D005767:Gastrointestinal Diseases / Q000453:epidemiology / Q000209:etiology; D006801:Humans; D007239:Infections / Q000453:epidemiology / Q000209:etiology; D053208:Kaplan-Meier Estimate; D008297:Male; D008527:Medulloblastoma / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000601:surgery / Q000628:therapy*; D018242:Neuroectodermal Tumors, Primitive / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000601:surgery / Q000628:therapy*; D036102:Peripheral Blood Stem Cell Transplantation; D016879:Salvage Therapy; D018805:Sepsis / Q000209:etiology / Q000401:mortality; D015173:Supratentorial Neoplasms / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy / Q000601:surgery / Q000628:therapy*; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D014750:Vincristine / Q000008:administration & dosage / Q000009:adverse effects
D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D002945:Cisplatin