Chen E L, Liu H L, Geng L Q, Tang B L, Zhu X Y, Yao W, Song K D, Wan X, Sun G Y, Qiang P, Fan Q, Zhou Z W, Zheng C C, Zhang L, Zhang X H, Tong J, Sun Z M
Anhui Provincial Hospital Affiliated to University of Science and Technology of China Institute of Hematology, Hefei 230001, China.
Zhonghua Xue Ye Xue Za Zhi. 2021 Jul 14;42(7):549-554. doi: 10.3760/cma.j.issn.0253-2727.2021.07.004.
To retrospectively analyze the clinical outcomes of single unrelated cord blood transplantation (UCBT) in children with high risk and refractory acute myeloid leukemia (AML) . Between June 2008 and December 2018, a total of 160 consecutive pediatric patients with AML received single UCBT (excluding acute promyelocytic leukemia) . Myeloablative conditioning (MAC) regimen were applied. All patients received a combination of cyclosporine A (CsA) and mycophenolate mofetil (MMF) for the prophylaxis of graft -versus- host disease (GVHD) . The cumulative incidence of neutrophil cells engraftment at day +42 and platelet recovery at day +120 was 95.0% (95% 90.0%-97.5%) at a median of 16 days after transplantation (range, 11-38 days) and 85.5% (95% 83.3%-93.4%) with a median time to recovery of 35 days (range, 13-158) , respectively. Incidence of grades Ⅱ-Ⅳ and Ⅲ-Ⅳ acute GVHD and chronic GVHD were 37.3% (95%CI 29.3%-45.2%) , 27.3% (95% 20.0%-35.0%) and 22.4% (95% 15.5%-28.7%) , respectively. The transplant-related mortality (TRM) at 360 day was 13.1% (95% 8.4%-18.9%) . The 5-year cumulative incidence of relapse was 13.8% (95% 8.5%-20.3%) . The 5-year disease-free survival (DFS) and overall survival (OS) were 71.7% (95% 62.7%-77.8%) and 72.2% (95% 64.1%-78.7%) , respectively. The 5-year GVHD and relapse free survival (GRFS) was 56.1% (95% 46.1%-64.9%) . The 5-year cumulative recurrence rates of CR1, CR2, and NR groups were 5.3%, 19.9%, and 30.9% (=0.001) , and the 5-year OS rates were 79.9% (95% 70.3%-86.7%) , 71.1% (95% 50.4%-84.4%) and 52.9% (95% 33.0%-69.3%) ((2)=7.552, =0.020) , respectively. For pediatric patients with high risk and refractory AML, UCBT is a safe and effective treatment option, and it is favorable to improve the survival rate in CR1 stage.
回顾性分析单份非血缘脐血移植（UCBT）治疗高危及难治性儿童急性髓系白血病（AML）的临床疗效。2008年6月至2018年12月，共有160例连续性儿童AML患者接受单份UCBT（不包括急性早幼粒细胞白血病）。采用清髓性预处理（MAC）方案。所有患者均接受环孢素A（CsA）和霉酚酸酯（MMF）联合预防移植物抗宿主病（GVHD）。移植后第42天中性粒细胞植入和第120天血小板恢复的累积发生率分别为95.0%（95% 90.0%-97.5%），移植后中位时间为16天（范围11-38天）和85.5%（95% 83.3%-93.4%），中位恢复时间为35天（范围13-158天）。Ⅱ-Ⅳ级和Ⅲ-Ⅳ级急性GVHD及慢性GVHD的发生率分别为37.3%（95%CI 29.3%-45.2%）、27.3%（95% 20.0%-35.0%）和22.4%（95% 15.5%-28.7%）。360天时移植相关死亡率（TRM）为13.1%（95% 8.4%-18.9%）。5年累积复发率为13.8%（95% 8.5%-20.3%）。5年无病生存率（DFS）和总生存率（OS）分别为71.7%（95% 62.7%-77.8%）和72.2%（95% 64.1%-78.7%）。5年无GVHD和复发生存率（GRFS）为56.1%（95% 46.1%-64.9%）。CR1、CR2和NR组的5年累积复发率分别为5.3%、19.9%和30.9%（P=0.001），5年OS率分别为79.9%（95% 70.3%-86.7%）、71.1%（95% 50.4%-84.4%）和52.9%（95% 33.0%-69.3%）（χ2=7.552，P=0.020）。对于高危及难治性AML儿童患者，UCBT是一种安全有效的治疗选择，有利于提高CR1期的生存率。
