Department of Pediatric and Adolescent Oncology, Institut Gustave Roussy, Villejuif, France.
Pediatr Blood Cancer. 2014 Jun;61(6):977-81. doi: 10.1002/pbc.24713. Epub 2013 Aug 23.
To evaluate long-term survival of the first cohort of stage-4 neuroblastoma patients treated with the N7 induction chemotherapy, surgery of the primary tumor and high-dose chemotherapy (HDC) containing Busulfan-Melphalan (Bu-Mel) followed by autologous stem cell transplantation (ASCT).
From 1998 to 1999, 47 children were included in the NB97 trial and treated with induction chemotherapy according to the N7 protocol, followed by surgery of the primary tumor. HDC (Busulfan, 600 mg/m(2) Melphalan, 140 mg/m(2) ) was administered in patients with partial response of metastases with no more than 3 mIBG spots. Radiotherapy was delivered to the primary tumor site when tumors displayed MYCN amplification.
Thirty-nine patients received Bu-Mel (83%): 23 who had achieved complete response (CR) of metastases, 20 after induction treatment and 3 after second-line chemotherapy, and 16 in partial response (PR). The toxicity of the whole treatment was manageable. The main HDC related-toxicity was hepatic veno-occlusive disease grade > 2 occurring in 15% of the patients. The 8-year EFS of the whole cohort was 34% (95% CI, 22-48%). The 8-year EFS of the 39 patients who received Bu-Mel and ASCT was 41% (95% CI, 27-57%). Patients who achieved a CR of metastases at the end of induction chemotherapy had a significantly better outcome than the others (8-year EFS, 52% vs. 20%; P = 0.02).
The long-term results of this first prospective cohort of patients with metastatic disease treated with the N7 induction chemotherapy and HDC (Bu-Mel) confirm published data with stable survival curves but with a longer follow-up.
评估接受 N7 诱导化疗、原发肿瘤手术和包含白消安-美法仑（Bu-Mel）的高剂量化疗（HDC）以及自体干细胞移植（ASCT）的 4 期神经母细胞瘤（NB）患者的第一队列的长期生存情况。
1998 年至 1999 年，NB97 试验纳入 47 名患儿，按照 N7 方案接受诱导化疗，随后进行原发肿瘤手术。对于转移灶有部分缓解（MR）且 131I-间碘苄胍（mIBG）摄取少于 3 个点的患者，给予 HDC（白消安 600mg/m2 ，美法仑 140mg/m2 ）。当肿瘤显示 MYCN 扩增时，给予原发肿瘤部位放疗。
39 名患者接受了 Bu-Mel（83%）治疗：23 名完全缓解（CR）的患者，20 名诱导治疗后缓解，3 名二线化疗后缓解，16 名部分缓解（PR）。整个治疗的毒性是可管理的。主要的 HDC 相关毒性是 15%的患者出现 2 级以上肝静脉闭塞性疾病。整个队列的 8 年 EFS 为 34%（95%CI，22-48%）。接受 Bu-Mel 和 ASCT 的 39 名患者的 8 年 EFS 为 41%（95%CI，27-57%）。诱导化疗结束时达到转移灶 CR 的患者的预后明显优于其他患者（8 年 EFS，52%比 20%；P=0.02）。
接受 N7 诱导化疗和 HDC（Bu-Mel）治疗的转移性疾病患者的这一前瞻性队列的长期结果证实了已有数据，其生存曲线稳定，但随访时间更长。
