两种不同预处理方案在高危神经母细胞瘤患儿自体移植前的比较。
Comparison of two different conditioning regimens before autologous transplantation for children with high-risk neuroblastoma.
机构信息
Hematology/Oncology Department, Bambino Gesù Children Hospital, Rome, Italy.
出版信息
Anticancer Res. 2012 Dec;32(12):5527-33.
BACKGROUND
Although high-dose chemotherapy (HDC) represents the standard of treatment for high-risk neuroblastoma (NBL), the most effective conditioning regimen still remains to be identified.
PATIENTS AND METHODS
Forty-one high-risk NBL entered into local protocol based on induction chemotherapy, surgery and HDC with either etoposide/thiotepa/cyclophophamide (ETC) or i.v. busulfan and L-PAM (Bu/L-PAM).
RESULTS
Thirty-seven patients underwent HDC; 29 with ETC and 8 with Bu/L-PAM. No toxic deaths were recorded. The 5-year progression-free survival (PFS) of patients given ETC was 21% (95% confidence interval CI (9-36%), while PFS for patients given Bu/L-PAM was 88% (95% CI=39-98%) (p<0.05). In multivariate analysis, treatment with the ETC regimen predicted progression/recurrence with a hazard ratio (HR) of 16.8 (p<0.05), as well as MYCN amplification which had an HR of 4.4 (p<0.05).
CONCLUSION
Although the number of studied cases is limited, our data suggest that in high-risk NBL the combination of Bu/L-PAM is superior to the ETC regimen.
背景
虽然大剂量化疗（HDC）是高危神经母细胞瘤（NBL）的标准治疗方法，但最有效的预处理方案仍有待确定。
患者和方法
41 例高危 NBL 患者按照诱导化疗、手术和 HDC 方案入组，预处理方案为依托泊苷/噻替哌/环磷酰胺（ETC）或静脉注射白消安和 L-PAM（Bu/L-PAM）。
结果
37 例患者接受了 HDC；ETC 预处理 29 例，Bu/L-PAM 预处理 8 例。无治疗相关死亡。接受 ETC 预处理的患者 5 年无进展生存率（PFS）为 21%（95%置信区间 CI 9-36%），而接受 Bu/L-PAM 预处理的患者 5 年 PFS 为 88%（95% CI=39-98%）（p<0.05）。多因素分析显示，ETC 方案治疗与进展/复发的风险比（HR）为 16.8（p<0.05），MYCN 扩增与 HR 为 4.4（p<0.05）。
结论
尽管研究病例数有限，但我们的数据表明，在高危 NBL 中，Bu/L-PAM 联合方案优于 ETC 方案。
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