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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(p OS)及无白血病存活率(p LFS)为72%及56%;其中标危AML患儿p LFS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p LFS为48%,化疗联合造血干细胞移植患儿p LFS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p LFS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。
Objective To investigate the efficacy and feasibility of NOPHO AML 2004 regimen and combined hematopoietic stem cell therapy in children with acute myeloid leukemia (AML). Methods A retrospective analysis was performed on pediatric patients with AML (no acute promyelocytic cells) aged <14 years treated with NOPHO AML 2004 (partial combined hematopoietic stem cell transplantation) from December 2009 to December 2015 in Nanfang Hospital, Southern Medical University. Leukemia) patients with clinical data, Kaplan Meier method for survival analysis and Log-Rank test. Results 53 cases of AML induced complete remission rate of 86.8%. Thirty-six patients (67.9%) completed 6 courses of chemotherapy and 14 (26.4%) received allogeneic hematopoietic stem cell transplantation after 2 to 3 courses of induction therapy. Three patients discontinued treatment after completing 1-3 courses of chemotherapy. The 5-year predicted total survival (p OS) and leukemia-free survival (p LFS) were 72% and 56%, respectively, for 12 to 82 months (median follow-up time 29 months) (P = 0.779). The p LFS in children with chemotherapy alone was 48%, and the p LFS in children with chemotherapy combined with hematopoietic stem cell transplantation was 77%. There was no significant difference between the two groups There was no significant difference between the two groups (P = 0.1268). On the 15th day, the 5-year p-value of bone marrow assessment, treatment response group (GR), middle treatment response group (IR), and treatment response poor group (PR) , 29%, 53% respectively. The difference between the two groups was statistically significant (P = 0.0071). Conclusion NOPHO AML 2004 is a feasible and effective clinical treatment for AML children in China. Day 15 bone marrow assessment of naive or naïve cells can be used as an important predictor of prognosis.