{"title":"接受酪氨酸激酶抑制剂治疗的慢性髓性白血病患者治疗失败的预测模型。","authors":"Xiaoshuai Zhang, Bingcheng Liu, Jian Huang, Yanli Zhang, Na Xu, Robert Peter Gale, Weiming Li, Xiaoli Liu, Huanling Zhu, Ling Pan, Yunfan Yang, Hai Lin, Xin Du, Rong Liang, Chunyan Chen, Xiaodong Wang, Guohui Li, Zhuogang Liu, Yanqing Zhang, Zhenfang Liu, Jianda Hu, Chunshui Liu, Fei Li, Wei Yang, Li Meng, Yanqiu Han, Li'e Lin, Zhenyu Zhao, Chuanqing Tu, Caifeng Zheng, Yanliang Bai, Zeping Zhou, Suning Chen, Huiying Qiu, Lijie Yang, Xiuli Sun, Hui Sun, Li Zhou, Zelin Liu, Danyu Wang, Jianxin Guo, Liping Pang, Qingshu Zeng, Xiaohui Suo, Weihua Zhang, Yuanjun Zheng, Xiaojun Huang, Qian Jiang","doi":"10.1182/blood.2024024761","DOIUrl":null,"url":null,"abstract":"<p><strong>Abstract: </strong>Although tyrosine kinase inhibitor (TKI) therapy has markedly improved the survival of people with chronic-phase chronic myeloid leukemia (CML), 20% to 30% of people still experienced therapy failure. Data from 1955 consecutive patients with chronic-phase CML diagnosed by the European LeukemiaNet recommendations from 1 center receiving initial imatinib or a second-generation (2G) TKI therapy were interrogated to develop a clinical prediction model for TKI-therapy failure. This model was subsequently validated in 3454 patients from 76 other centers. Using the predictive clinical covariates associated with TKI-therapy failure, we developed a model that stratified patients into low-, intermediate- and high-risk subgroups with significantly different cumulative incidences of therapy failure (P < .001). There was good discrimination and calibration in the external validation data set, and the performance was consistent with that of the training data set. Our model had the better prediction discrimination than the Sokal and European Treatment and Outcome Study long-term survival scores, with the greater time-dependent area under the receiver-operator characteristic curve values and a better ability to redefine the risk of therapy failure. Our model could help physicians estimate the likelihood of initial imatinib or 2G TKI-therapy failure in people with chronic-phase CML.</p>","PeriodicalId":9102,"journal":{"name":"Blood","volume":null,"pages":null},"PeriodicalIF":21.0000,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11551847/pdf/","citationCount":"0","resultStr":"{\"title\":\"A predictive model for therapy failure in patients with chronic myeloid leukemia receiving tyrosine kinase inhibitor therapy.\",\"authors\":\"Xiaoshuai Zhang, Bingcheng Liu, Jian Huang, Yanli Zhang, Na Xu, Robert Peter Gale, Weiming Li, Xiaoli Liu, Huanling Zhu, Ling Pan, Yunfan Yang, Hai Lin, Xin Du, Rong Liang, Chunyan Chen, Xiaodong Wang, Guohui Li, Zhuogang Liu, Yanqing Zhang, Zhenfang Liu, Jianda Hu, Chunshui Liu, Fei Li, Wei Yang, Li Meng, Yanqiu Han, Li'e Lin, Zhenyu Zhao, Chuanqing Tu, Caifeng Zheng, Yanliang Bai, Zeping Zhou, Suning Chen, Huiying Qiu, Lijie Yang, Xiuli Sun, Hui Sun, Li Zhou, Zelin Liu, Danyu Wang, Jianxin Guo, Liping Pang, Qingshu Zeng, Xiaohui Suo, Weihua Zhang, Yuanjun Zheng, Xiaojun Huang, Qian Jiang\",\"doi\":\"10.1182/blood.2024024761\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Abstract: </strong>Although tyrosine kinase inhibitor (TKI) therapy has markedly improved the survival of people with chronic-phase chronic myeloid leukemia (CML), 20% to 30% of people still experienced therapy failure. Data from 1955 consecutive patients with chronic-phase CML diagnosed by the European LeukemiaNet recommendations from 1 center receiving initial imatinib or a second-generation (2G) TKI therapy were interrogated to develop a clinical prediction model for TKI-therapy failure. This model was subsequently validated in 3454 patients from 76 other centers. Using the predictive clinical covariates associated with TKI-therapy failure, we developed a model that stratified patients into low-, intermediate- and high-risk subgroups with significantly different cumulative incidences of therapy failure (P < .001). There was good discrimination and calibration in the external validation data set, and the performance was consistent with that of the training data set. Our model had the better prediction discrimination than the Sokal and European Treatment and Outcome Study long-term survival scores, with the greater time-dependent area under the receiver-operator characteristic curve values and a better ability to redefine the risk of therapy failure. Our model could help physicians estimate the likelihood of initial imatinib or 2G TKI-therapy failure in people with chronic-phase CML.</p>\",\"PeriodicalId\":9102,\"journal\":{\"name\":\"Blood\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":21.0000,\"publicationDate\":\"2024-10-31\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11551847/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Blood\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1182/blood.2024024761\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1182/blood.2024024761","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
A predictive model for therapy failure in patients with chronic myeloid leukemia receiving tyrosine kinase inhibitor therapy.
Abstract: Although tyrosine kinase inhibitor (TKI) therapy has markedly improved the survival of people with chronic-phase chronic myeloid leukemia (CML), 20% to 30% of people still experienced therapy failure. Data from 1955 consecutive patients with chronic-phase CML diagnosed by the European LeukemiaNet recommendations from 1 center receiving initial imatinib or a second-generation (2G) TKI therapy were interrogated to develop a clinical prediction model for TKI-therapy failure. This model was subsequently validated in 3454 patients from 76 other centers. Using the predictive clinical covariates associated with TKI-therapy failure, we developed a model that stratified patients into low-, intermediate- and high-risk subgroups with significantly different cumulative incidences of therapy failure (P < .001). There was good discrimination and calibration in the external validation data set, and the performance was consistent with that of the training data set. Our model had the better prediction discrimination than the Sokal and European Treatment and Outcome Study long-term survival scores, with the greater time-dependent area under the receiver-operator characteristic curve values and a better ability to redefine the risk of therapy failure. Our model could help physicians estimate the likelihood of initial imatinib or 2G TKI-therapy failure in people with chronic-phase CML.
期刊介绍:
Blood, the official journal of the American Society of Hematology, published online and in print, provides an international forum for the publication of original articles describing basic laboratory, translational, and clinical investigations in hematology. Primary research articles will be published under the following scientific categories: Clinical Trials and Observations; Gene Therapy; Hematopoiesis and Stem Cells; Immunobiology and Immunotherapy scope; Myeloid Neoplasia; Lymphoid Neoplasia; Phagocytes, Granulocytes and Myelopoiesis; Platelets and Thrombopoiesis; Red Cells, Iron and Erythropoiesis; Thrombosis and Hemostasis; Transfusion Medicine; Transplantation; and Vascular Biology. Papers can be listed under more than one category as appropriate.