We aimed to develop a concise objectifiable risk evaluation (CORE) tool for predicting non-relapse mortality (NRM) and overall survival (OS) after allogeneic hematopoietic stem cell transplantation (allo-HCT). A total of 1120 adult patients who had undergone allo-HCT at our center between 2013 and 2020 were divided into training, first, and second validation cohorts. Objectifiable, patient-related factors impacting NRM in univariate and multivariate analyses were: serum albumin, serum creatinine, serum C-reactive protein (CRP), heart function (LVEF), lung function (VC, FEV1), and patient age. Hazard ratios were assigned points (0–3) based on their impact on NRM and summed to the individual CORE HCT score. The CORE HCT score stratified patients into three distinct low-, intermediate-, and high-risk groups with two-year NRM rates of 9%, 22%, and 46%, respectively, and OS rates of 73%, 55%, and 35%, respectively (p< 0.001). These findings were confirmed in a first and a second recently treated validation cohort. Importantly, the CORE HCT score remained informative across various conditioning intensities, disease-specific subgroups, and donor types, but did not impact relapse incidence. A comparison of CORE HCT vs. HCT Comorbidity Index (HCT-CI) in the second validation cohort revealed better performance of the CORE HCT score with c-statistics for NRM and OS of 0.666 (SE 0.05,p= 0.001) and 0.675 (SE 0.039,p< 0.001) vs. 0.431 (SE 0.057,p= 0.223) and 0.535 (SE 0.042,p= 0.411), respectively. The CORE HCT score is a concise and objectifiable risk evaluation tool for adult patients undergoing allo-HCT for malignant disease. External multicenter validation is underway.
本研究旨在开发一种用于预测异基因造血干细胞移植（allo-HCT）后非复发死亡率（NRM）和总生存期（OS）的简明客观化风险评估（CORE）工具。我们纳入了2013年至2020年间在本中心接受allo-HCT的1120例成年患者，并将其分为训练队列、第一验证队列和第二验证队列。通过单因素和多因素分析，确定影响NRM的客观化患者相关因素包括：血清白蛋白、血清肌酐、血清C反应蛋白（CRP）、心脏功能（左心室射血分数，LVEF）、肺功能（肺活量VC，第一秒用力呼气容积FEV1）以及患者年龄。根据各因素对NRM的影响程度分配风险比对应的分值（0-3分），并累加得到个体CORE HCT评分。CORE HCT评分将患者分为低危、中危和高危三个明显不同的风险组，其两年NRM率分别为9%、22%和46%，OS率分别为73%、55%和35%（p<0.001）。这些发现在第一及第二近期治疗验证队列中均得到证实。重要的是，CORE HCT评分在不同预处理强度、疾病特异性亚组和供者类型中均具有预测价值，但不影响复发率。在第二验证队列中，CORE HCT评分与HCT合并症指数（HCT-CI）的比较显示，CORE HCT评分性能更优，其预测NRM和OS的c统计值分别为0.666（标准误0.05，p=0.001）和0.675（标准误0.039，p<0.001），而HCT-CI的相应值分别为0.431（标准误0.057，p=0.223）和0.535（标准误0.042，p=0.411）。CORE HCT评分是一种适用于因恶性疾病接受allo-HCT的成年患者的简明、客观化风险评估工具。目前正在进行外部多中心验证。
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