Objective: To evaluate the efficacy of decitabine combined with low dose chemotherapy (LDC) in the treatment of high-risk, refractory and relapsed pediatric acute myeloid leukemia (AML). Methods: Clinical data of 19 AML children treated with decitabine combined with LDC in the Department of Hematology, Children's Hospital of Soochow University from April 2017 to November 2019 were analyzed retrospectively. The therapeutic response, adverse effects and survival status were analyzed,and the outcomes of patients were followed up. Results: Among 19 AML cases, there were 10 males and 9 females. Five cases were high-risk AML, 7 cases were refractory AML, and 7 cases were relapsed AML. After one course of decitabine+LDC treatment, 15 cases achieved complete remission, 3 cases got partial remission, and only 1 case didn't get remission. All patients received allogeneic hematopoietic stem cell transplantation as consolidation therapy. The follow-up time of all cases was 46 (37, 58) months, 14 children had survived. The cumulative three-year overall survival rate was (79±9) %, events free survival rates was (68±11) %, and recurrence free survival rate was (81±10) %. The most common adverse effects related to the induction treatment were cytopenia (19 cases) and infection (16 cases).There were no treatment-related death during the therapy. Conclusion: Decitabine combined with LDC is a safe and effective option for high-risk, refractory and relapsed AML children, which provides an opportunity for HSCT.
目的： 评价地西他滨联合低剂量化疗治疗高危、难治、复发儿童急性髓系白血病（AML）的效果。 方法： 回顾性分析苏州大学附属儿童医院血液科2017年4月至2019年11月接受地西他滨联合低剂量化疗的19例AML患儿的临床资料，分析治疗反应、治疗相关不良反应和生存情况，并随访患儿结局。 结果： 19例AML患儿中男10例、女9例；高危5例、难治7例、复发7例。19例患儿经1轮地西他滨+LDC治疗后达到完全缓解15例，部分缓解3例，未缓解1例。19例患儿均接受了异基因造血干细胞移植作为巩固治疗。19例患儿随访46（37，58）个月，存活14例、死亡5例。3年累计总生存率为（79±9）%，无事件生存率为（68±11）%，无复发生存率为（81±10）%。诱导治疗后常见的不良反应是血细胞减少（19例）和感染（16例），无治疗相关死亡病例。 结论： 地西他滨联合低剂量化疗是治疗高危、难治、复发儿童AML安全、有效的治疗方案，为患儿进行异基因造血干细胞移植创造了机会。.