Laprie Anne, Michon Jean, Hartmann Olivier, Munzer Caroline, Leclair Marc-David, Coze Carole, Valteau-Couanet Dominique, Plantaz Dominique, Carrie Christian, Habrand Jean-Louis, Bergeron Christophe, Chastagner Pascal, Défachelles Anne-Sophie, Delattre Olivier, Combaret Valérie, Bénard Jean, Pérel Yves, Gandemer Virginie, Rubie Hervé
Département d'Hémato-Oncologie, Hôpital des Enfants, Toulouse, France.
Cancer. 2004 Sep 1;101(5):1081-9. doi: 10.1002/cncr.20453.
The objective of this study was to determine whether systemic and regional, intensified treatment can improve the outcome of children who present with a localized neuroblastoma (NB) with MYCN amplification (MNA).
Between 1990 and 2000, 610 children with localized NB were included in the Localized Neuroblastoma 90 (NBL 90) and NBL 94 study from the French Society of Pediatric Oncology. Among them, 32 children had MNA with Stage II or III NB. During the first period of the study, 20 children (Group A) received postoperative conventional chemotherapy (CT) and/or radiotherapy (RT), depending on each patient's postoperative status. Subsequently, because of a high recurrence rate, the next 12 children (Group B) were given postoperative high-dose CT (HDC) (busulfan and melphalan) with stem cell rescue (SCR) followed by RT in addition to conventional postoperative CT.
The two groups were comparable with regard to prognostic factors (age, location of the primary lesion, International Neuroblastoma Staging System stage, lymph node invasion) and response to preoperative CT. The 6-year overall survival rate was significantly different between the two groups 25% +/- 10% in Group A vs. 83% +/- 11% in Group B; P = 0.004).
Postoperative intensification treatment with HDC, SCR, and locoregional RT resulted in higher survival rates when compared with standard treatment alone and should be considered in the treatment plan for children who are diagnosed with Stage II or III NB and MYCN amplification.
本研究的目的是确定全身和局部强化治疗是否能改善患有MYCN基因扩增（MNA）的局限性神经母细胞瘤（NB）患儿的预后。
1990年至2000年期间，法国儿科肿瘤学会的局限性神经母细胞瘤90（NBL 90）和NBL 94研究纳入了610例局限性NB患儿。其中，32例患儿患有MNA且为Ⅱ期或Ⅲ期NB。在研究的第一阶段，20例患儿（A组）根据每位患者的术后状态接受术后常规化疗（CT）和/或放疗（RT）。随后，由于复发率高，接下来的12例患儿（B组）接受了术后大剂量CT（HDC）（白消安和美法仑）联合干细胞救援（SCR），除常规术后CT外还进行了RT。
两组在预后因素（年龄、原发灶位置、国际神经母细胞瘤分期系统分期、淋巴结侵犯）和对术前CT的反应方面具有可比性。两组的6年总生存率有显著差异，A组为25%±10%，B组为83%±11%；P = 0.004）。
与单纯标准治疗相比，术后采用HDC、SCR和局部区域RT的强化治疗可提高生存率，对于诊断为Ⅱ期或Ⅲ期NB且有MYCN扩增的患儿，治疗方案中应考虑采用该治疗方法。
