异基因造血干细胞移植治疗急性髓系白血病伴骨髓增生异常相关改变75例临床分析
目的:评价异基因造血干细胞移植（allo-HSCT）治疗急性髓系白血病伴骨髓增生异常相关改变（AML-MRC）的疗效及预后因素，分析AML-MRC患者基因突变谱系并探讨影响移植预后的分子生物学特征。方法:对2006年至2020年于中国医学科学院血液病医院接受allo-HSCT的75例AML-MRC患者进行回顾性分析。将75例患者分为：H组[既往有骨髓增生异常综合征（MDS）或MDS/骨髓增殖性肿瘤（MPN）病史]、C组（新诊断的AML-MRC伴MDS相关细胞遗传学异常）和M组（新诊断的AML-MRC伴多系发育异常），分析三组患者临床特征差异以及对移植预后的影响。对43例患者行骨髓靶向二代测序（...
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| Published in | 中华血液学杂志 Vol. 42; no. 10; pp. 814 - 822 |
|---|---|
| Main Authors | , , , , , , , , , , , |
| Format | Journal Article |
| Language | Chinese |
| Published |
中国医学科学院、北京协和医学院血液病医院（中国医学科学院血液学研究所），实验血液学国家重点实验室，国家血液系统疾病临床医学研究中心，天津 300020
01.10.2021
|
| Subjects | |
| Online Access | Get full text |
| ISSN | 0253-2727 |
| DOI | 10.3760/cma.j.issn.0253-2727.2021.10.004 |
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| Abstract | 目的:评价异基因造血干细胞移植（allo-HSCT）治疗急性髓系白血病伴骨髓增生异常相关改变（AML-MRC）的疗效及预后因素，分析AML-MRC患者基因突变谱系并探讨影响移植预后的分子生物学特征。方法:对2006年至2020年于中国医学科学院血液病医院接受allo-HSCT的75例AML-MRC患者进行回顾性分析。将75例患者分为：H组[既往有骨髓增生异常综合征（MDS）或MDS/骨髓增殖性肿瘤（MPN）病史]、C组（新诊断的AML-MRC伴MDS相关细胞遗传学异常）和M组（新诊断的AML-MRC伴多系发育异常），分析三组患者临床特征差异以及对移植预后的影响。对43例患者行骨髓靶向二代测序（137个基因）。结果:①75例AML-MRC患者，男41例，女34例，中位年龄41（18~56）岁，中位随访时间为35（95%
CI 30~49）个月，中位总生存（OS）时间为78个月（95%
CI 23个月~未达到）。移植后3年OS率为57.1%（95%
CI 45.6%~71.4%），无事件生存（EFS）率为52.0%（95%
CI 40.8%~66.1%），累积复发率（CIR）为26.8%（95%
CI 16.6%~30.0%），移植相关死亡率（TRM）为22.7%（95%
CI 13.2%~33.8%）。多因素分析显示，移植前处于非第1次完全缓解（CR1）状态是移植后OS和EFS的独立危险因素。影响OS的独立危险因素还包括-5/5q-染色体核型异常、移植后未发生慢性移植物抗宿主病（慢性GVHD）。②75例患者中H组59例（78.7%），其中20例转化为白血病（转白）前曾接受去甲基化药物治疗；C组9例（12.0%），M组7例（9.3%）。M、H、C组移植后3年OS率分别为71.4%（95%
CI 44.7%~100.0%）、55.0%（95%
CI 41.8%~72.5%）、55.6%（95%
CI 31.0%~99.7%）（
P=0.700），EFS率分别为71.4%（95%
CI 44.7%~100.0%）、46.5%（95%
CI 34.0%~63.8%）、55.6%（95%
CI 31.0%~99.7%）（
P=0.600）；原发性AML-MRC与继发性AML-MRC相比，移植后3年OS、EFS率差异无统计学意义[61.9%（95%
CI 41.9%~91.4%）对55.0%（95%
CI 41.8%~72.5 |
|---|---|
| AbstractList | 目的:评价异基因造血干细胞移植（allo-HSCT）治疗急性髓系白血病伴骨髓增生异常相关改变（AML-MRC）的疗效及预后因素，分析AML-MRC患者基因突变谱系并探讨影响移植预后的分子生物学特征。方法:对2006年至2020年于中国医学科学院血液病医院接受allo-HSCT的75例AML-MRC患者进行回顾性分析。将75例患者分为：H组[既往有骨髓增生异常综合征（MDS）或MDS/骨髓增殖性肿瘤（MPN）病史]、C组（新诊断的AML-MRC伴MDS相关细胞遗传学异常）和M组（新诊断的AML-MRC伴多系发育异常），分析三组患者临床特征差异以及对移植预后的影响。对43例患者行骨髓靶向二代测序（137个基因）。结果:①75例AML-MRC患者，男41例，女34例，中位年龄41（18~56）岁，中位随访时间为35（95%
CI 30~49）个月，中位总生存（OS）时间为78个月（95%
CI 23个月~未达到）。移植后3年OS率为57.1%（95%
CI 45.6%~71.4%），无事件生存（EFS）率为52.0%（95%
CI 40.8%~66.1%），累积复发率（CIR）为26.8%（95%
CI 16.6%~30.0%），移植相关死亡率（TRM）为22.7%（95%
CI 13.2%~33.8%）。多因素分析显示，移植前处于非第1次完全缓解（CR1）状态是移植后OS和EFS的独立危险因素。影响OS的独立危险因素还包括-5/5q-染色体核型异常、移植后未发生慢性移植物抗宿主病（慢性GVHD）。②75例患者中H组59例（78.7%），其中20例转化为白血病（转白）前曾接受去甲基化药物治疗；C组9例（12.0%），M组7例（9.3%）。M、H、C组移植后3年OS率分别为71.4%（95%
CI 44.7%~100.0%）、55.0%（95%
CI 41.8%~72.5%）、55.6%（95%
CI 31.0%~99.7%）（
P=0.700），EFS率分别为71.4%（95%
CI 44.7%~100.0%）、46.5%（95%
CI 34.0%~63.8%）、55.6%（95%
CI 31.0%~99.7%）（
P=0.600）；原发性AML-MRC与继发性AML-MRC相比，移植后3年OS、EFS率差异无统计学意义[61.9%（95%
CI 41.9%~91.4%）对55.0%（95%
CI 41.8%~72.5 |
| Abstract_FL | Objective:To evaluate the outcomes and prognostic factors of adults with acute myeloid leukemia with myelodysplastic-related changes (AML-MRC) who received allogeneic hematopoietic stem cell transplantation (allo-HSCT) . The genetic mutation lineage of patients with AML-MRC and the molecular mutation affecting the transplantation prognosis was discussed.Methods:The clinical data of 75 patients with AML-MRC who underwent allo-HACT from 2006 to 2020 were retrospectively analyzed for clinical characteristics, survival, relapse-related indicators, and risk factors affecting transplantation prognosis. Additionally, the clinical characteristics and prognosis of multilineage dysplasia (M) group, history of myelodysplastic syndrome (MDS) or myelodysplastic syndrome/myelodysplastic proliferative tumor (MDS/MPN) (H) group, and MDS related cytogenetic abnormalities (C) group were compared. The bone marrow of 43 patients underwent targeting second-generation sequencing (137 genes) .Results:①There were 41 males and 34 females with a median age of 41 (18-56) years, a median follow-up time of 35 (95%
CI 30-49) months, and a median survival time (OS) of 78 (95%
CI 23-) months. Three-year OS and event-free survival (EFS) were 57.1% (95%
CI 45.6%-71.4%) and 52.0% (95%
CI 40.8%-66.1%) . Also, the three-year cumulative recurrence rate (CIR) and transplant-related mortality rate (TRM) were 26.8% (95%
CI 16.6%-30.0%) and 22.7% (95%
CI 13.2%-33.8%) , respectively. Furthermore, multivariate analysis revealed that pre-transplant non-CR1 status was an independent risk factor for OS and EFS. Other independent risk factors for OS included abnormal karyotype of -5/5q- chromosome and the absence of chronic graft-versus-host disease (cGVHD) after transplantation. ②Among the 75 patients, 59 (78.7%) were in group H, 20 had received demethylation drugs before turning to AML and nine cases (12.0%) in group C and seven cases (9.3%) in group M. There was no significant difference in the three-year OS and EFS among the three groups[group M
vs H
vs C: OS: 71.4% (95%
CI 44.7%-100.0%)
vs 55.0% (95%
CI 41.8%-72.5%)
vs 55.6% (95%
CI 31.0%-99.7%) ,
P=0.700; EFS: 71.4% (95%
CI 44.7%-100.0%)
vs 46.5% (95%
CI 34.0%-63.8%)
vs 55.6% (95%
CI 31.0%-99.7%) ,
P=0.600]. Compared with primary and secondary AML-MRC, there was no statistically significant difference in the three-year OS and EFS[61.9% (95%
CI 41.9%-91.4%)
vs 55.0% (95%
CI 41.8%-72.5%) ,
P=0.600; 61.9% (95%
CI 41.9%-91.4%)
vs 46.5% (95%
CI 34.0%-63.8%) ,
P=0.400]. Furthermore, there was no significant difference in the time to AML between patients who received demethylation treatment before (20 cases) and those who did not (39 cases) [195 (16-937) d
vs 162 (9-3167) d,
P=0.804]. Moreover, there were no statistically significant differences in the three-year OS and EFS between the two groups (
P=0.400,
P=0.700) . ③ NGS test was performed on bone marrow samples of 43 patients (57.3%) , and 73 mutation types were found. Additionally, U2AF1 had the highest mutation incidence (11 cases, 25.6%) , and more than 10% were found: RUNX1 (ten cases, 23.3%) , NRAS (ten cases, 23.3%) , ASXL1 (six cases, 14.0%) , PTPN11 (five cases, 11.6%) , TET2 (five cases, 11.6%) . Univariate analysis showed U2AF1[
P=0.875,
HR=1.110 (95%
CI 0.295-4.195) ], RUNX1[
P=0.685,
HR=0.728 (95%
CI 0.157-3.375) ], NRAS[
P=0.919,
HR=0.923 (95%
CI 0.196-4.334) ] mutation did not affect OS.
Conclusion:Chromosome abnormality of -5/5q-, cGVHD, and non-CR1 status before transplantation were independent risk factors for OS in patients with allo-HSCT and AML-MRC. Additionally, the MHC subgroup classification was not a factor affecting the prognosis of transplantation. Treatment with demethylated drugs may not delay MDS turning to AML and prolong the OS after transplantation. |
| Author | 张海啸 张荣莉 何祎 杨栋林 翟卫华 马巧玲 陈欣 庞爱明 冯四洲 韩明哲 姜尔烈 魏嘉璘 |
| AuthorAffiliation | 中国医学科学院、北京协和医学院血液病医院（中国医学科学院血液学研究所），实验血液学国家重点实验室，国家血液系统疾病临床医学研究中心，天津 300020 |
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| Author_FL | Zhai Weihua Wei Jialin Zhang Rongli Chen Xin Ma Qiaoling He Yi Yang Donglin Han Mingzhe Pang Aiming Zhang Haixiao Feng Sizhou Jiang Erlie |
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| Author_xml | – sequence: 1 fullname: 张海啸 – sequence: 2 fullname: 庞爱明 – sequence: 3 fullname: 陈欣 – sequence: 4 fullname: 张荣莉 – sequence: 5 fullname: 翟卫华 – sequence: 6 fullname: 马巧玲 – sequence: 7 fullname: 杨栋林 – sequence: 8 fullname: 魏嘉璘 – sequence: 9 fullname: 何祎 – sequence: 10 fullname: 冯四洲 – sequence: 11 fullname: 韩明哲 – sequence: 12 fullname: 姜尔烈 |
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| Keywords | Acute myeloid leukemia with myelodysplastic related changes 急性髓系白血病伴骨髓增生异常相关改变 Genetic mutations 造血干细胞移植 基因突变 Hematopoietic stem cell transplantation |
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| Publisher | 中国医学科学院、北京协和医学院血液病医院（中国医学科学院血液学研究所），实验血液学国家重点实验室，国家血液系统疾病临床医学研究中心，天津 300020 |
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| Title | 异基因造血干细胞移植治疗急性髓系白血病伴骨髓增生异常相关改变75例临床分析 |
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