[TP53异常的骨髓增生异常综合征/急性髓系白血病患者接受异基因造血干细胞移植的生存疗效]
[Survival efficacy of MDS/AML patients with TP53 abnormal received allogeneic hematopoietic stem cell transplantation].
作者信息
Feng D, Wang M Y, Liu J, Zhang H X, Chen X, Zhang R L, Zhai W H, Ma Q L, Pang A M, Yang D L, Wei J L, He Y, Feng S Z, Han M Z, Jiang E L
机构信息
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
出版信息
Zhonghua Xue Ye Xue Za Zhi. 2023 Mar 14;44(3):222-229. doi: 10.3760/cma.j.issn.0253-2727.2023.03.008.
TP53-abnormal MDS/acute myeloid leukemia (AML) patients' allogeneic hematopoietic stem cell transplantation (allo-HSCT) treatment's effectiveness and influencing factors should be studied. 42 patients with TP53 gene status change MDS/AML who underwent allo-HSCT from 2014.8.1 to 2021.7.31 at the Hematology Hospital of the Chinese Academy of Medical Sciences were the subject of a retrospective analysis. The 42 patients were divided into three groups: the TP53 deletion group (group A) , TP53 mono-alle mutation group (group B) , and TP53 multi-hit group (group C) . The differences in clinical features and prognostic factors after transplantation were analyzed. There were 42 MDS/AML patients, including 21 patients with MDS, and 21 patients with AML. The median follow-up period was 34.0 (7.5-75.0) months and the median patient age at the time of transplantation was 41.5 (18-63) years old. The total OS was 66.3% (95% 53.4%-82.4%) in 3 years after transplantation, and EFS was 61.0% (95% 47.7%-78.0%) in 3 years. For 3 years after receiving hematopoietic stem cell transplantation, there were no statistically significant differences in 3-year OS and EFS in groups A, B, and C (≥0.05) . The 3 years OS was 82.5% (95% 63.1%-100.0%) in group A, 60.6% (95% 43.5%-84.4%) in group B, and 57.1% (95% 30.1%-100.0%) in group C. Univariate analysis revealed that the number of co-mutant genes, pre-HSCT treatment, and disease type did not affect prognosis, while age, karyotype, co-mutation, positive blast cell before transplantation, and positive blast cell after transplantation were common prognostic factors for OS and EFS (<0.1) . MRD levels before transplantation were found to be independent risk factors for OS (=0.037, =33.40, 95% 1.24-901.17) in a multivariate analysis. Patients with MDS/AML who have TP53 mutations can benefit from allo-HSCT, but patients with complex karyotypes have a worse prognosis. Meanwhile, the final flow cytometry (FCM) monitoring blast cell test before HSCT has a certain guiding significance for prognostic assessment.
应研究TP53异常的骨髓增生异常综合征/急性髓系白血病（MDS/AML）患者异基因造血干细胞移植（allo-HSCT）治疗的有效性及影响因素。对2014年8月1日至2021年7月31日在中国医学科学院血液病医院接受allo-HSCT的42例TP53基因状态改变的MDS/AML患者进行回顾性分析。将42例患者分为三组：TP53缺失组（A组）、TP53单等位基因突变组（B组）和TP53多打击组（C组）。分析移植后临床特征及预后因素的差异。42例MDS/AML患者中，MDS患者21例，AML患者21例。中位随访时间为34.0（7.5 - 75.0）个月，移植时患者中位年龄为41.5（18 - 63）岁。移植后3年总生存率（OS）为66.3%（95% 53.4% - 82.4%），无事件生存率（EFS）为61.0%（95% 47.7% - 78.0%）。造血干细胞移植后3年，A、B、C三组的3年OS和EFS差异无统计学意义（≥0.05）。A组3年OS为82.5%（95% 63.1% - 100.0%），B组为60.6%（95% 43.5% - 84.4%），C组为57.1%（95% 30.1% - 100.0%）。单因素分析显示，共突变基因数量、移植前治疗及疾病类型不影响预后，而年龄、核型、共突变、移植前原始细胞阳性及移植后原始细胞阳性是OS和EFS的常见预后因素（<0.1）。多因素分析发现移植前微小残留病（MRD）水平是OS的独立危险因素（=0.037，=33.40，95% 1.24 - 901.17）。TP53突变的MDS/AML患者可从allo-HSCT中获益，但核型复杂的患者预后较差。同时，HSCT前最终流式细胞术（FCM）监测原始细胞检测对预后评估有一定指导意义。
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