文章：

一种新型经爱荷华–梅奥验证的异基因干细胞移植生存结局预测复合风险评估工具

A novel Iowa–Mayo validated composite risk assessment tool for allogeneic stem cell transplantation survival outcome prediction

原文发布日期：2021-11-20

DOI: 10.1038/s41408-021-00573-6

类型: Article

开放获取: 是

英文摘要：

Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative option for many hematologic conditions and is associated with considerable morbidity and mortality. Therefore, prognostic tools are essential to navigate the complex patient, disease, donor, and transplant characteristics that differentially influence outcomes. We developed a novel, comprehensive composite prognostic tool. Using a lasso-penalized Cox regression model (n = 273), performance status, HCT-CI, refined disease-risk index (rDRI), donor and recipient CMV status, and donor age were identified as predictors of disease-free survival (DFS). The results for overall survival (OS) were similar except for recipient CMV status not being included in the model. Models were validated in an external dataset (n = 378) and resulted in a c-statistic of 0.61 and 0.62 for DFS and OS, respectively. Importantly, this tool incorporates donor age as a variable, which has an important role in HSCT outcomes. This needs to be further studied in prospective models. An easy-to-use and a web-based nomogram can be accessed here: https://allohsctsurvivalcalc.iowa.uiowa.edu/.
 

摘要翻译： 

异基因造血干细胞移植（HSCT）是多种血液系统疾病的根治性治疗手段，但其伴随显著的发病率和死亡率。因此，预后评估工具对于整合患者、疾病、供者和移植特征等复杂因素对预后的差异性影响至关重要。我们开发了一种新型的综合预后评估工具。通过套索惩罚Cox回归模型（n=273），研究确认体能状态、HCT合并症指数、改良疾病风险指数（rDRI）、供受者巨细胞病毒血清学状态以及供者年龄是无病生存期（DFS）的预测因素。总生存期（OS）的预测模型结果相似，但未纳入受者巨细胞病毒状态。模型在外部队列（n=378）中验证，DFS和OS的C统计量分别为0.61和0.62。值得注意的是，该工具首次将供者年龄作为影响HSCT预后的关键变量纳入评估体系，这一点需在前瞻性模型中进一步验证。用户可通过以下链接访问便捷的在线列线图工具：https://allohsctsurvivalcalc.iowa.uiowa.edu/。

原文链接：

A novel Iowa–Mayo validated composite risk assessment tool for allogeneic stem cell transplantation survival outcome prediction