Sung Ki Woong, Yoo Keon Hee, Cho Eun Joo, Koo Hong Hoe, Lim Do Hoon, Shin Hyung Jin, Ahn Seung Do, Ra Young Shin, Choi Eun Seok, Ghim Thad T
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
Pediatr Blood Cancer. 2007 Apr;48(4):408-15. doi: 10.1002/pbc.21064.
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.
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.
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.
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.
采用单次或串联双次大剂量化疗（HDCT）治疗新诊断的高危或复发的髓母细胞瘤及幕上原始神经外胚层肿瘤（MB/sPNET）患儿，以推迟或避免年幼儿童接受放疗。
对25例新诊断的高危或复发的MB/sPNET患儿进行了37次HDCT治疗。最初打算接受双次HDCT的15例患者中有12例采用了串联双次HDCT。
6例新诊断的高危（>3岁）、8例新诊断的（<3岁）和11例复发的MB/sPNET患者的3年无事件生存率（±标准误）分别为83.3±15.2%、62.5±20.5%和29.1±15.7%。首次HDCT时处于完全缓解（CR）或部分缓解（PR）以及未达到PR的患者的3年无事件生存率分别为67.4±11.0%和16.7±15.2%（P = 0.001）。最初打算接受双次HDCT和单次HDCT的患者的3年无事件生存率分别为66.0±12.4%和40.0±15.5%。对于首次HDCT时处于CR或PR的19例患者，串联双次HDCT组和单次HDCT组的3年无事件生存率分别为88.9±10.5%和44.4±16.6%（P = 0.037）。虽然在25次首次HDCT治疗期间发生了4例治疗相关死亡（TRM），但在12次第二次HDCT治疗期间未发生TRM。8例年幼儿童中有4例成功避免了全脑全脊髓放疗且随后未复发。
大剂量化疗可能提高新诊断的高危MB/sPNET患儿的生存率，并在一定程度上提高复发的MB/sPNET患儿的生存率。有必要进一步研究以阐明串联双次HDCT的疗效。
