Stram D O, Matthay K K, O'Leary M, Reynolds C P, Haase G M, Atkinson J B, Brodeur G M, Seeger R C
Department of Preventive Medicine, University of Southern California School of Medicine, Los Angeles, USA.
J Clin Oncol. 1996 Sep;14(9):2417-26. doi: 10.1200/JCO.1996.14.9.2417.
To compare event-free survival (EFS) for patients with stage IV neuroblastoma who were treated with induction chemotherapy followed by additional courses of the same chemotherapy or by intensive chemoradiotherapy and autologous bone marrow transplantation (ABMT).
Two hundred seven children who were diagnosed with stage IV neuroblastoma after 1 year of age were given five to seven courses of induction chemotherapy consisting of cisplatin, etoposide, doxorubicin, and cyclophosphamide (CCC-321-P2). This chemotherapy was continued for 13 total courses for some patients, whereas intensive chemoradiotherapy with ABMT was given to others (CCG-321-P3). The decision to continue chemotherapy versus to consolidate with chemoradiotherapy was not randomized but was made by parents and physicians. Marrow used for ABMT was purged ex vivo and was free of immunocytologically detectable neuroblastoma cells.
One hundred fifty-nine of 207 patients (77%) remained event-free during induction therapy. Of these, 67 received chemoradiotherapy/ABMT (CCG-321-P3) and 74 continued chemotherapy (CCG-321-P2). Using Cox regression analysis, the relative risk (RR) of an event after chemoradiotherapy/ABMT was estimated to be 58% of that for patients who continued chemotherapy (P = .01). Similarly, Kaplan-Meier analysis estimated EFS at four years for the chemoradiotherapy/ABMT and chemotherapy groups to be 40% and 19% respectively (P = .019). Subgroups appearing to benefit from chemoradiotherapy/ABMT were those with only a partial tumor response to induction chemotherapy (RR = 0.43; P = .008; EFS, 29% v 6%) and those whose tumors had amplification of the N-myc gene (RR = 0.26; P = .112; EFS, 67% v 0%).
Consolidation with intensive, myeloablative chemoradiotherapy followed by purged ABMT may be more effective than continuing chemotherapy for patients with stage IV neuroblastoma.
比较接受诱导化疗后继续相同化疗疗程或接受强化放化疗及自体骨髓移植（ABMT）的IV期神经母细胞瘤患者的无事件生存期（EFS）。
207例1岁后被诊断为IV期神经母细胞瘤的儿童接受了5至7个疗程的诱导化疗，化疗方案为顺铂、依托泊苷、阿霉素和环磷酰胺（CCC - 321 - P2）。部分患者继续进行总共13个疗程的该化疗，而其他患者则接受强化放化疗及ABMT（CCG - 321 - P3）。继续化疗还是采用放化疗巩固治疗的决定并非随机做出，而是由家长和医生决定。用于ABMT的骨髓在体外进行了净化，且无免疫细胞检测可发现的神经母细胞瘤细胞。
207例患者中有159例（77%）在诱导治疗期间无事件发生。其中，67例接受了放化疗/ABMT（CCG - 321 - P3），74例继续化疗（CCG - 321 - P2）。使用Cox回归分析，放化疗/ABMT后发生事件的相对风险（RR）估计为继续化疗患者的58%（P = 0.01）。同样，Kaplan - Meier分析估计放化疗/ABMT组和化疗组4年的EFS分别为40%和19%（P = 0.019）。似乎从放化疗/ABMT中获益的亚组包括那些对诱导化疗仅有部分肿瘤反应的患者（RR = 0.43；P = 0.008；EFS，29%对6%）以及肿瘤有N - myc基因扩增的患者（RR = 0.26；P = 0.112；EFS，67%对0%）。
对于IV期神经母细胞瘤患者，采用强化的清髓性放化疗随后进行净化的ABMT巩固治疗可能比继续化疗更有效。
