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用于预测接受异基因造血干细胞移植的慢性粒-单核细胞白血病患者早期复发的多因素风险评分系统:一项全国代表性多中心研究

A multifactorial risk scoring system for the prediction of early relapse in CMML patients with allo-HSCT: a nationwide representative multicenter study.

作者信息

Zhou Jian-Ying, Chen Yu-Xiu, Yuan Hai-Long, Xu Ya-Jing, Huang Xiao-Bing, Gao Su-Jun, Zhang Yi-Cheng, Zhou Fang, Song Xian-Min, Luo Yi, Yang Jian-Min, Li Yu-Hua, Wang Shun-Qing, Dong Yu-Jun, Zhang Xi, Feng Yi-Mei, Du Xin, Zhu Han, Zhu Zun-Min, Bi Ke-Hong, Jiang Ming, Niu Ting, Wan Ding-Ming, Chen Yi, Liu Li, Yi Hai, Chen Yu-Hong, Wang Feng-Rong, Zhang Yuan-Yuan, Mo Xiao-Dong, Han Wei, Wang Jing-Zhi, Wang Yu, Chen Huan, Zhao Xiang-Yu, Chang Ying-Jun, Liu Kai-Yan, Huang Xiao-Jun, Zhang Xiao-Hui

机构信息

Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.

Department of Hematology, The First Affiliated Hospital of Xinjiang Medical University, Urumqi, China.

出版信息

Bone Marrow Transplant. 2025 Mar;60(3):310-318. doi: 10.1038/s41409-024-02480-3. Epub 2024 Nov 25.

Abstract

Chronic myelomonocytic leukemia (CMML) is a clonal hematopoietic stem cell malignancy and the only curable therapy is allogeneic hematopoietic stem cell transplantation (allo-HSCT). However, allo-HSCT is not appropriate for all CMML patients, and relapse is the leading cause of treatment failure. This project conducted a nationwide multicenter real-world study to develop a novel prediction scoring system for early relapse. A total of 238 CMML patients from twenty-seven medical centers treated with allo-HSCT, and 307 adult patients with CMML who underwent allo-HSCT in a publicly available research dataset from the Center for International Blood and Marrow Transplantation Registry (CIBMTR) database were included. Independent prognostic factors for the early relapse of CMML posttransplantation were identified according to competing risk regression methods. Four prognostic factors were identified: bone marrow blasts >10% (hazard ratio [HR], 4.262; P = 0.014), age >60 years (HR, 6.221; P = 0.007), hemoglobin level <100 g/L (HR, 3.695; P = 0.004), and non TET2 gene mutation (HR, 3.425; P = 0.017). A risk-grading scoring system was developed based on the regression coefficients and patients were stratified into low-risk (0-1 point), intermediate-risk (1.5-2 points) and high-risk ( > 2 points) groups. The validated internal c-statistic was 0.767 (95% confidence interval [CI], 0.674-0.860), and the external c-statistic was 0.769 (95% CI, 0.703-0.836). In the derivation cohort, the cumulative incidence rates of early relapse in the low-risk, intermediate-risk, and high-risk groups were 1.35% (95% CI: 1-4%), 10.40% (95% CI: 4-16%), and 29.54% (95% CI: 16-39%) (P < 0.001), respectively. This scoring system can be utilized to early identification of patients at a high risk of relapse and contributing to the implementation of urgent medical support.

摘要

慢性粒单核细胞白血病(CMML)是一种克隆性造血干细胞恶性肿瘤,唯一可治愈的疗法是异基因造血干细胞移植(allo-HSCT)。然而,allo-HSCT并不适用于所有CMML患者,复发是治疗失败的主要原因。本项目开展了一项全国多中心真实世界研究,以开发一种用于早期复发的新型预测评分系统。纳入了来自27个医学中心接受allo-HSCT治疗的238例CMML患者,以及国际血液和骨髓移植研究中心(CIBMTR)数据库公开研究数据集中接受allo-HSCT的307例成年CMML患者。根据竞争风险回归方法确定CMML移植后早期复发的独立预后因素。确定了四个预后因素:骨髓原始细胞>10%(风险比[HR],4.262;P = 0.014)、年龄>60岁(HR,6.221;P = 0.007)、血红蛋白水平<100 g/L(HR,3.695;P = 0.004)和非TET2基因突变(HR,3.425;P = 0.017)。基于回归系数开发了一种风险分级评分系统,患者被分为低风险(0 - 1分)、中风险(1.5 - 2分)和高风险(>2分)组。验证后的内部c统计量为0.767(95%置信区间[CI],0.674 - 0.860),外部c统计量为0.769(95% CI,0.703 - 0.836)。在推导队列中,低风险、中风险和高风险组的早期复发累积发生率分别为1.35%(95% CI:1 - 4%)、10.40%(95% CI:4 - 16%)和29.54%(95% CI:16 - 39%)(P < 0.001)。该评分系统可用于早期识别复发风险高的患者,并有助于实施紧急医疗支持。

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