联合社会人口学因素及临床因素预测初发慢性髓性白血病慢性期患者治疗反应及结局  

Combination of socio-demographic and clinical co-variates for predicting treatment responses and outcomes in patients with chronic myeloid leukemia in the chronic phase

作　　者：张小帅 秦亚溱[1] 赖悦云[1] 石红霞[1] 黄晓军[1] 江倩[1] Zhang Xiaoshuai;Qin Yazhen;Lai Yueyun;Shi Hongxia;Huang Xiaojun;Jiang Qian(Peking University People's Hospital,Peking University Institute of Hematology,National Clinical Research Center for Hematologic Disease,Beijing 100044,China)

机构地区：[1]北京大学人民医院、北京大学血液病研究所、国家血液系统疾病临床医学研究中心

出　　处：《中华血液学杂志》2022年第1期54-62,共9页Chinese Journal of Hematology

基　　金：国家自然科学基金(81770161、81970140)。

摘　　要：目的探讨影响初发慢性髓性白血病慢性期(CML-CP)治疗反应及结局的社会人口学因素及临床因素,并联合社会人口学因素及临床因素分别建立针对治疗反应及结局新的预测模型。方法回顾性分析2006年1月至2020年12月在北京大学人民医院确诊的≥18岁初诊接受酪氨酸激酶抑制剂(TKI)一线治疗、具有完整社会人口学及临床资料的CML-CP连续病例。应用Cox回归模型探索治疗反应和结局的独立影响因素,治疗反应包括完全细胞遗传学反应(CCyR)、主要分子学反应(MMR)、分子学反应4(MR^(4))、分子学反应4.5(MR^(4.5)),结局包括无失败生存(FFS)、无进展生存(PFS)、总生存(OS)、CML相关生存(CML-OS)。结果研究共纳入1414例以伊马替尼(1176例)、尼洛替尼(170例)或达沙替尼(68例)为一线治疗的成人CML-CP患者,中位年龄40(18~83)岁,男性873例(61.7%)。多因素分析显示,受教育水平低(P值<0.001~0.070)和ELTS评分中/高危(P值<0.001~0.009)是较低的细胞遗传学、分子学反应获得率以及各种不良治疗结局的共同影响因素。此外,男性、农村户籍以及初诊时WBC≥120×10^(9)/L、HGB<115 g/L和一线伊马替尼治疗与较低的细胞遗传学反应和(或)分子学反应获得率均显著相关;单身、离异或丧偶、农村户籍以及初诊时WBC≥120×10^(9)/L、HGB<115 g/L和伴有合并症与较低的FFS、PFS、OS和(或)CML-OS率显著相关。联合社会人口学因素及临床因素分别建立针对治疗反应、治疗失败及疾病进展以及生存的预测模型,根据上述预测模型将整体患者分为各风险亚组,亚组间治疗反应及结局差异均具有统计学意义(P值均<0.001)。结论除临床因素外,社会人口学因素与CML-CP患者治疗反应及结局密切相关。联合社会人口学因素与临床特征可较好预测患者的治疗反应及结局。Objective To explore the impacts of socio-demographic and clinical co-variates on treatment responses and outcomes in patients with chronic myeloid leukemia in the chronic phase(CML-CP)receiving tyrosine kinase inhibitor(TKI)and identified the predictive models for them.Methods Data of newly diagnosed adult patients with CML-CP receiving first-line TKI and having complete socio-demographic data and clinical information were reviewed.Cox model was used to identify the independent variables associated with complete cytogenetic response(CCyR),major molecular response(MMR),molecular response 4(MR^(4))and molecular response 4.5(MR^(4.5)),as well as failure-free survival(FFS),progression-free survival(PFS),overall survival(OS)and CML-related OS.Results A total of 1414 CML-CP patients treated with first-line imatinib(n=1176),nilotinib(n=170)or dasatinib(n=68)were reviewed.Median age was 40(18-83)years and 873 patients(61.7%)were males.Result of the multivariate analysis showed that lower educational level(P<0.001-0.070)and EUTOS long-term survival intermediate or high-risk(P<0.001-0.009)were significantly associated with lower cumulative incidences of CCyR,MMR,MR4 and MR4.5,as well as the inferior FFS,PFS,OS and CML-related OS.In addition,those who were males,from rural households,had white blood cells(WBC)≥120×10^(9)/L,hemoglobin(HGB)<115 g/L and treated with first-line imatinib had significantly lower cumulative incidences of cytogenetic and/or molecular responses.Being single,divorced or widowed,having,rural household registration,WBC≥120×10^(9)/L,HGB<15 g/L,and comorbidity(ies)was significantly associated with inferior FFS,PFS,OS,and/or CML-related OS.Thereafter,the patients were classified into several subgroups using the socio-demographic characteristics and clinical variables by cytogenetic and molecular responses,treatment failure and disease progression,as well as overall survival and CML-related OS,respectively.There were significant differences in treatment responses and outcomes among the subgroups(P<0

关 键 词：白血病 髓系 慢性 治疗结果 社会学因素 临床因素 

分 类 号：R733.72[医药卫生—肿瘤]