Abstract:

Objective　To analyze the effectiveness and the practicability of Nordic Society for Pediatric Hematology and Oncology（NOPHO） AML 2004 protocol combined with hematopoietic stem cell transplantation（HSCT） in treating childhood acute myeloid leukemia（AML） in China. Methods　The clinical data of 53 children（aged under 14） with AML（not including acute promyelocytic leukemia） treated with NOPHO AML 2004 protocol（some children followed by HSCT） between Dec. 2009 and Dec. 2015 was retrospectively analyzed. Survival was evaluated by Kaplan Meier analysis and Log-Rank test. Results　Complete remission（CR） rate reached 86.8%. Totally 36 patients（67.9%） finished 6 courses of chemotherapy；14 patients（26.4%）underwent HSCT after 1-3 courses of consolidation chemotherapy；3 patients gave up treatment after 1-3 courses of chemotherapy. The proleptic overall survival（pOS） at 5 years and leukemia-free survival（pLFS） was 72% and 56% respectively during a follow-up period of 12 to 82 months（median 29 months）. For standard-risk patients，the pLFS at 5 years reached 58%， while high-risk patients had 46%（P＝0.779）. The pLFS at 5 years in patients who only underwent chemotherapy and those with additional hematopoietic stem cell transplantation was 48% and 77% respectively（P＝0.1268）. The pLFS in patients with good response，intermediate response and poor response on day 15 bone marrow examination was 75%，29% and 53% respectively（P＝0.0071）. Conclusion　NOPHO AML 2004 protocol is effective and feasible for Chinese patients with childhood AML. The evaluation of blast in bone marrow on day 15 can be used as an important prognostic indicator.

Key words: acute myeloid leukemia, chemotherapy, child, hematopoietic stem cell transplantation

摘要：

目的    探讨NOPHO AML 2004方案及联合造血干细胞治疗儿童急性髓系白血病（AML）的疗效及可行性。方法　回顾性分析南方医科大学南方医院儿科2009年12月至2015年12月按计划完成NOPHO AML 2004方案（部分联合造血干细胞移植）治疗的年龄＜14岁的儿童AML（不含急性早幼粒细胞性白血病）患者的临床资料，采用Kaplan Meier方法进行生存分析及Log-Rank检验。结果　53例AML诱导完全缓解率达86.8%。36例（67.9%）完成6个疗程化疗，14例（26.4%）在2～3个疗程诱导治疗后行异基因造血干细胞移植，3例在完成1～3个疗程化疗后放弃治疗。随访12～82个月（中位随访时间29个月），5年预计总存活率（pOS）及无白血病存活率（pLFS）为72%及56%；其中标危AML患儿pLFS为58%，高危患儿46%，两组间差异无统计学意义（P＝0.779）；单纯化疗患儿pLFS为48%，化疗联合造血干细胞移植患儿pLFS为77%，两组间差异无统计学意义（P＝0.1268）； 第15天骨髓评估， 治疗反应好组（GR）、中等治疗反应组（IR）、 治疗反应差组（PR）患儿5年pLFS分别为75%、 29%、 53%，组间差异有统计学意义（P＝0.0071）。结论　NOPHO AML 2004方案治疗中国AML患儿可行且有较好的临床疗效。第15天骨髓评估原始或幼稚细胞可作为预测预后重要指标。

关键词: 急性非淋巴细胞性白血病, 化疗, 儿童, 造血干细胞移植