Zhang Jian-Hua, Zhang Ao-Li, Dong Chun-Xia, Qin Xiao-Qi, Zhang Rui-Juan, Ge Xiao-Yan, Kang Jian-Min, Hou Yan-Fei, Zhang Yao-Fang, Guo Jian-Li, Yang Lin-Hua
Second Clinical College, Shanxi Metical University; Department of Hematology, The Second Hospital of Shanxi Medical University, Taiyuan 030001, Shanxi Province, China.
Second Clinical College, Shanxi Metical University; Department of Hematology, The Second Hospital of Shanxi Medical University, Taiyuan 030001, Shanxi Province, China,E-mail:
Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2019 Apr;27(2):360-364. doi: 10.19746/j.cnki.issn.1009-2137.2019.02.008.
To investigate the safety and efficacy of autologous peripheral blood hematopoietic stem cell transplantation (auto-PBHSCT) using modified BU/CY conditioning regimen for young AML patients of low and middle risk in the first complete remission (CR1).
Ten young AML patients of low and middle risk who did not want to accept allogeneic hematopoietic stem cell transplantation（allo-HSCT）and underwent auto-PBHSCT in CR1 during May 2013 to December 2016 were retrospectively analyzed. From 3 months after auto-PBHSCT, the maintenance therapy with interleukin-2 (IL-2) or IL-2 combined with histamine dihydrochloride was performed for these patients in the next 18 months. The side effects of the conditioning regimen, hematopoietic recovery time, transplant-related mortality (TRM) within 100 days and 1 year after auto-PBHSCT, relapse rate, leukemia-free survival (LFS) rate at 2 years and 3 years, overall survival (OS) were evaluated at 3 years and 4 years.
Gastrointestinal side effects were the major non-hematologic toxicity reaction, among which, 7 cases relatively mild and 3 cases displayed moderate, just one case suffered from severe reaction. In 4 cases, the mild liver damage occurred, but no hemorrhagic cystitis occurred. All the patients experienced different kinds of infection, including 5 cases of bloodstream infection, 2 cases of gastrointestinal infection, 3 cases of crissum infection and 2 cases of oral infection. The myeloablative effect occurred in all ten patients. The median times for absolute neutrophil count (ANC)<0.5×10/L and for platelet count <20.0×10/L were 1.5 (0-3) days and 3 (2-5) days after transplantation, respectively. The patients achieved ANC>0.5×10/L at 10 to 19 days, median was 13 days after auto-PBHSCT. The patients achieved platelet count >20×10/L at 10 to 72 days; median was 32 days after auto-PBHSCT. The TRM within 100 days and 1 year after transplantation was 0. The relapse occurred in 2 cases at 6 and 14 months after auto-PBHSCT raspectively. The median follow-up time was 48.1 months, and the median survival time was 54.7 months after transplantation. The 2-year and 3-year LFS were 100% (10 cases) and 80% (8 cases), respectively. The 3-year and 4-year OS were 80% (8 cases) and 70% (7 cases), respectively.
Modified BU/CY as conditioning regimen for auto-PBHSCT can achieve the myeloablative effect without raising TRM and obtain good LFS and OS. As for young AML patients without high risk, it is a valuable therapeutic option, especially for those lacking the chance of allo-HSCT.
探讨改良BU/CY预处理方案用于低中危年轻急性髓系白血病（AML）患者首次完全缓解期（CR1）自体外周血造血干细胞移植（auto-PBHSCT）的安全性和疗效。
回顾性分析2013年5月至2016年12月期间10例低中危年轻AML患者，这些患者不愿接受异基因造血干细胞移植（allo-HSCT），于CR1期接受了auto-PBHSCT。自auto-PBHSCT后3个月起，对这些患者在接下来的18个月内进行白细胞介素-2（IL-2）或IL-2联合盐酸组胺的维持治疗。评估预处理方案的副作用、造血恢复时间、auto-PBHSCT后100天及1年内的移植相关死亡率（TRM）、复发率、2年和3年无白血病生存率（LFS）、3年和4年总生存率（OS）。
胃肠道副作用是主要的非血液学毒性反应，其中7例较轻微，3例为中度，仅1例为重度反应。4例出现轻度肝损害，未发生出血性膀胱炎。所有患者均发生了不同类型的感染，包括5例血流感染、2例胃肠道感染、3例肛周感染和2例口腔感染。10例患者均出现了清髓效果。移植后中性粒细胞绝对值计数（ANC）<0.5×10⁹/L和血小板计数<20.0×10⁹/L的中位时间分别为1.5（0-3）天和3（2-5）天。患者在auto-PBHSCT后10至19天达到ANC>0.5×10⁹/L，中位时间为13天。患者在10至72天达到血小板计数>20×10⁹/L，中位时间为auto-PBHSCT后32天。移植后100天及1年内的TRM为0。auto-PBHSCT后分别于6个月和14个月有2例复发。中位随访时间为48.1个月，移植后中位生存时间为54.7个月。2年和3年LFS分别为100%（10例）和80%（8例）。3年和4年OS分别为80%（8例）和70%（7例）。
改良BU/CY作为auto-PBHSCT的预处理方案可实现清髓效果且不增加TRM，并获得良好的LFS和OS。对于无高危因素的年轻AML患者，尤其是那些缺乏allo-HSCT机会的患者，是一种有价值的治疗选择。
