Comparison of a fludarabine and melphalan combination-based reduced toxicity conditioning with myeloablative conditioning by radiation and/or busulfan in acute myeloid leukemia in Japanese children and adolescents.
Comparison of a fludarabine and melphalan combination-based reduced toxicity conditioning with myeloablative conditioning by radiation and/or busulfan in acute myeloid leukemia in Japanese children and adolescents.
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日本儿童和青少年急性髓系白血病中基于氟达拉滨和美法仑组合的减毒调理与放射和/或白消安清髓调理的比较。
DOI：
10.1002/pbc.25389
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发表时间：
2015
影响因子：
3.2
通讯作者：
Kudo K
中科院分区：
文献类型：
--
作者：
Ishida H;Adachi S;Hasegawa D;Okamoto Y;Goto H;Inagaki J;Inoue M;Koh K;Yabe H;Kawa K;Kato K;Atsuta Y;Kudo K
急性髓系白血病（AML）患儿减毒预处理（RTC）后异基因造血细胞移植（allo‐HCT）与标准清髓预处理（MAC）的相对疗效尚未得到广泛研究。为了解决RTC是否是缓解期儿童AML患者的可行方法，我们使用2000年至2011年在日本收集的全国注册数据，对RTC或标准MAC后接受allo-HCT的患者首次移植的结局进行了回顾性调查。程序我们比较了基于氟达拉滨（Flu）和美法仑（Mel）的方案（RTC; n = 34），全身照射（TBI）和/或基于白消安（Bu）的预处理（MAC; n = 102）在第一次或第二次完全缓解（CR 1/CR2）的人口统计学和疾病标准匹配的儿童和青少年AML患者中。和慢性GVHD在各预处理组中相似。复发风险（25% vs. 26%）和非复发死亡率3年后的生存率（13% vs. 11%）在这些组之间没有差异，单变量和多变量分析表明，Flu/Mel‐RTC和MAC后的3年总生存率（OS）相当（平均值，72% [范围，51-85%]和68% [范围，58-77%]，分别）.ConclusionsThe结果表明，流感/梅尔-RTC方案是一个临床上可接受的预处理策略，为儿童和青少年AML患者在缓解。虽然这种回顾性的、基于登记的分析有一些局限性，但RTC值得在前瞻性试验中进一步研究。儿科血液癌症2015;62：883-889。© 2014 Wiley Periodicals，Inc.
BackgroundThe relative efficacy of allogeneic hematopoietic cell transplantation (allo‐HCT) after reduced toxicity conditioning (RTC) compared with standard myeloablative conditioning (MAC) in pediatric patients with acute myeloid leukemia (AML) has not been studied extensively. To address whether RTC is a feasible approach for pediatric patients with AML in remission, we performed a retrospective investigation of the outcomes of the first transplant in patients who had received an allo‐HCT after RTC or standard MAC, using nationwide registration data collected between 2000 and 2011 in Japan.ProcedureWe compared a fludarabine (Flu) and melphalan (Mel)‐based regimen (RTC; n = 34) with total body irradiation (TBI) and/or busulfan (Bu)‐based conditioning (MAC; n = 102) in demographic‐ and disease‐criteria‐matched childhood and adolescent patients with AML in first or second complete remission (CR1/CR2).ResultsThe incidence of engraftment, early complications, grade II–IV acute graft‐versus‐host disease (GVHD), and chronic GVHD were similar in each conditioning group. The risk of relapse (25%vs. 26%) and non‐relapse mortality (13%vs. 11%) after 3 years did not differ between these groups, and univariate and multivariate analyses demonstrated that the 3‐year overall survival (OS) rates after Flu/Mel‐RTC and MAC were comparable (mean, 72% [range, 51–85%] and 68% [range, 58–77%], respectively).ConclusionsThe results suggest that the Flu/Mel‐RTC regimen is a clinically acceptable conditioning strategy for childhood and adolescent patients with AML in remission. Although this retrospective, registry‐based analysis has several limitations, RTC deserves to be further investigated in prospective trials. Pediatr Blood Cancer 2015;62:883–889. © 2014 Wiley Periodicals, Inc.