Department of Hematology and Oncology, Shanghai Children's Hospital, Shanghai Jiao Tong University, No. 24, lane 1400, West Beijing Road, Shanghai 200040, China.
World J Pediatr. 2020 Apr;16(2):152-158. doi: 10.1007/s12519-019-00321-8. Epub 2019 Nov 20.
The prognosis for relapsed or refractory acute myeloid leukemia (RR-AML) in children is poor, and the preferred salvage chemotherapy is unclear. One regimen is cladribine, cytarabine, and granulocyte-colony stimulating factor (CLAG), but little is known about its efficacy and safety in children with RR-AML.
We enrolled RR-AML patients aged 0-18 years who received modified CLAG regimen for re-induction between July 1, 2015 and April 1, 2018, or conventional induction between August 1, 2011 and April 1, 2018. Patients were followed up to March 31, 2019. Patients underwent allogeneic stem cell transplantation (allo-SCT) or chemotherapy after the induction of complete remission (CR). The CR rate, survival, and side effects were analyzed.
The CR rate for induction was 66.7% after one cycle and 75.0% after two cycles of the CLAG regimen in 12 children. The nine children who received conventional chemotherapy had a CR rate of 22.2% after one cycle and 33.3% after two cycles (P = 0.087 vs. CLAG). The 3-year event-free survival (EFS) of the CLAG group and the conventional treatment group were 44.4 ± 15.7% and 22.2 ± 13.8% (P = 0.112). The 3-year overall survival of the two groups were 59.5 ± 16.2% and 22.2% ± 13.8% (P = 0.057). The 3-year EFS for allo-SCT and chemotherapy after CLAG regimen was 66.7 ± 19.2% and 25.0 ± 21.7% (P = 0.015). A single case of chemotherapy-related death was recorded.
Our data suggest a promising CR rate using CLAG salvage treatment in childhood RR-AML. Allo-SCT after CR may improve the long-term outcome in these patients.
儿童复发或难治性急性髓系白血病(RR-AML)的预后较差,首选的挽救性化疗方案尚不明确。其中一种方案是克拉屈滨、阿糖胞苷和粒细胞集落刺激因子(CLAG),但对于接受 RR-AML 治疗的儿童,其疗效和安全性知之甚少。
我们纳入了 2015 年 7 月 1 日至 2018 年 4 月 1 日期间接受改良 CLAG 方案或 2011 年 8 月 1 日至 2018 年 4 月 1 日期间接受常规诱导治疗的 0-18 岁 RR-AML 患者。患者随访至 2019 年 3 月 31 日。诱导缓解后患者行异基因造血干细胞移植(allo-SCT)或化疗。分析 CR 率、生存情况和不良反应。
12 例患者接受 CLAG 方案治疗,1 个疗程后 CR 率为 66.7%,2 个疗程后为 75.0%。9 例接受常规化疗的患者,1 个疗程后 CR 率为 22.2%,2 个疗程后为 33.3%(P=0.087 与 CLAG 相比)。CLAG 组和常规治疗组的 3 年无事件生存(EFS)率分别为 44.4±15.7%和 22.2±13.8%(P=0.112)。两组的 3 年总生存率分别为 59.5±16.2%和 22.2%±13.8%(P=0.057)。CLAG 方案治疗后行 allo-SCT 和化疗的患者 3 年 EFS 率分别为 66.7±19.2%和 25.0±21.7%(P=0.015)。仅记录到 1 例化疗相关死亡。
我们的数据表明,CLAG 挽救治疗在儿童 RR-AML 中具有较高的 CR 率。CR 后行 allo-SCT 可能改善这些患者的长期预后。