Zage Peter E, Kletzel Morris, Murray Kevin, Marcus Robert, Castleberry Robert, Zhang Yang, London Wendy B, Kretschmar Cynthia
Children's Cancer Hospital, The University of Texas M.D. Anderson Cancer Center, Houston, Texas, USA.
Pediatr Blood Cancer. 2008 Dec;51(6):747-53. doi: 10.1002/pbc.21713.
From 1993 to 1995, the Pediatric Oncology Group (POG) enrolled patients with high-risk neuroblastoma on three sequential, conjoined studies: a phase II induction window (9340), followed by intensive multiagent induction chemotherapy (9341), and subsequent myeloablative therapy with autologous stem cell rescue (9342). We report here the outcomes of patients treated on these studies.
Patients were between 1 and 21 years old with high-risk neuroblastoma. Phase II window therapy consisted of two courses of either paclitaxel, topotecan, or cyclophosphamide with topotecan. Induction therapy consisted of at least five cycles of intensive chemotherapy, followed by myeloablative therapy with purged autologous stem cell reinfusion. Patient responses, treatment toxicities, and overall and event-free survival rates were calculated.
Eighty-four percent of patients responded to induction chemotherapy, with 39% achieving complete response. Toxicities were primarily hematologic. The 7-year EFS and OS rates for all eligible patients on POG 9341 were 23 +/- 4% and 28 +/- 4%, respectively. The 7-year EFS and OS rates for patients treated on POG 9342 were 27 +/- 6% and 29 +/- 6%, respectively.
These studies were the first attempt by POG to use autologous stem cell transplantation for neuroblastoma treatment in a cooperative group setting. Toxicities and outcomes were comparable to contemporary cooperative group studies. The phase II induction window had no detectable effect on outcomes. New strategies are needed to improve survival for this devastating disease.
1993年至1995年,儿科肿瘤学组(POG)在三项连续的联合研究中纳入了高危神经母细胞瘤患者:一个II期诱导窗口研究(9340),随后是强化多药诱导化疗(9341),以及随后的自体干细胞救援清髓性治疗(9342)。我们在此报告这些研究中治疗患者的结果。
患者年龄在1至21岁之间,患有高危神经母细胞瘤。II期窗口治疗包括两疗程的紫杉醇、拓扑替康,或环磷酰胺与拓扑替康联合治疗。诱导治疗包括至少五个周期的强化化疗,随后是经净化的自体干细胞回输清髓性治疗。计算患者的反应、治疗毒性以及总生存率和无事件生存率。
84%的患者对诱导化疗有反应,39%达到完全缓解。毒性主要为血液学毒性。POG 9341中所有符合条件患者的7年无事件生存率(EFS)和总生存率(OS)分别为23±4%和28±4%。POG 9342中治疗患者的7年EFS和OS分别为27±6%和29±6%。
这些研究是POG在合作组环境中首次尝试使用自体干细胞移植治疗神经母细胞瘤。毒性和结果与当代合作组研究相当。II期诱导窗口对结果没有可检测到的影响。需要新的策略来提高这种毁灭性疾病的生存率。