【摘要】 目的 探讨减低化疗剂量的改良CLAG方案(克拉屈滨、阿糖胞苷、粒细胞集落刺激因子)治疗复发/难治急性髓系白血病(R/R-AML)儿童的疗效及安全性。方法 回顾性分析2016年6月至2023年4月接受改良CLAG方案治疗的R/R-AML患儿的临床资料，计算总体反应率(ORR)、不良反应发生率、总生存(OS)率和无事件生存(EFS)率。结果 26例患儿中复发17例，其中1例为睾丸白血病复发，3例为骨髓2次复发，余均为骨髓首次复发，难治9例。所有患儿均完成1疗程改良CLAG方案化疗，1例未评估治疗反应桥接造血干细胞移植，余25例患儿ORR为84.0 %(21/25)。复发患儿的ORR为81.3 %(13/16)，难治患儿的ORR为88.9 %(8/9)。细胞遗传学分层为低危的患儿ORR为76.9 %(10/13)，中高危患儿ORR为91.7 %(11/12)。所有患儿64个月的OS率和EFS率分别为69.7 %和63.3 %，15例治疗有反应并顺利桥接异基因造血干细胞移植的患儿64个月OS率和EFS率均为92.3 %。最常见的不良反应为骨髓抑制(100 %)和胃肠道反应(100 %)，其次为感染(57.7 %)、转氨酶升高(34.6 %)、出血(19.2 %)，1例患儿因4级颅内出血放弃，其他不良反应均经对症治疗后好转。结论 减低化疗剂量的改良CLAG方案是儿童R/R-AML的一种有效、安全的治疗选择。更多还原

【Abstract】 Objective To investigate the efficacy and safety of modified CLAG regimen (cladribine + cytarabine + granulocyte colony-stimulating factor) with reduced chemotherapy dose in the treatment of children with relapsed/refractory acute myeloid leukemia (R/R-AML).Methods The clinical data of children with R/R-AML treated with the modified CLAG regimen from June 2016 to April 2023 were retrospectively analyzed,and the overall response rate (ORR),incidence of adverse reactions,overall survival (OS) rate,and event-free survival (EFS) rate were calculated.Results Among all the children,17 had recurrent AML,of them 1 had testicular leukemia recurrence,3 had secondary recurrence in the bone marrow,and the rest had first recurrence in the bone marrow.Nine patients had refractory AML.All children completed 1 course of chemotherapy with modified CLAG regimen,and hematopoietic stem cell transplantation (HSCT) was bridged in 1 patient with no assessed response to treatment,with an ORR of 84.0 % (21/25) in the remaining 25 children.The ORR was 81.3 % (13/16) in the relapsed children and 88.9 % (8/9) in the refractory children.The ORR was 76.9 % (10/13) in children with low risk cytogenetic stratification and 91.7 % (11/12) in children with moderate and high risk cytogenetic stratification.The 64-month OS and EFS rates of all patients were 69.7 % and 63.3 %,respectively.The 64-month OS and EFS rates of 15 children who responded to treatment and successfully bridged allogeneic HSCT were 92.3 %.The most common adverse reactions were myelosuppression (100 %) and gastrointestinal reactions (100 %),followed by infection (57.7 %),elevated transaminases (34.6 %),and bleeding (19.2 %),and one child abandoned treatment due to grade 4 intracranial hemorrhage.All other adverse reactions were improved after symptomatic treatment.Conclusions The modified CLAG regimen with reduced chemotherapy dose is an effective and safe treatment option for children with R/R-AML.更多还原