Fattinger Nicolas, Roth Jan A, Baldomero Helen, Stolz Daiana, Medinger Michael, Heim Dominik, Tamm Michael, Halter Jörg P, Passweg Jakob R, Kleber Martina
Divisions of Hematology, University Hospital Basel, Switzerland.
University of Basel, Switzerland.
Hemasphere. 2022 Mar 11;6(4):e704. doi: 10.1097/HS9.0000000000000704. eCollection 2022 Apr.
Pretransplant risk scores such as the revised Pretransplant Assessment of Mortality (rPAM) score help to predict outcome of patients receiving allogeneic hematopoietic cell transplantation (allo-HCT). Since the rPAM has not been validated externally in a heterogeneous patient population with different diseases, we aimed to validate the rPAM score in a real-world cohort of allo-HCT patients. A total of 429 patients were included receiving their first allo-HCT from 2008 to 2015. The predictive capacity of the rPAM score for 4-year overall survival (OS), nonrelapse mortality (NRM), and cumulative incidence of relapse (CIR) after allo-HCT was evaluated. Moreover, we evaluated the impact of the rPAM score for OS and used uni- and multivariable analyses to identify patient- and transplant-related predictors for OS. In rPAM score categories of <17, 17-23, 24-30, and >30, the OS probability at 4 years differed significantly with 61%, 36%, 26%, and 10%, respectively ( < 0.0001). In contrast to CIR, the NRM increased significantly in patients with higher rPAM scores ( < 0.001). Regarding the OS, the rPAM score had an area under the receiver operating characteristics curve of 0.676 (95% confidence interval [CI], 0.625-0.727) at 4 years. In the multivariable analysis, the rPAM score was associated with OS-independently of conditioning regimens (adjusted hazard ratio per 1-unit increase, 1.10; 95% CI, 1.06-1.10; < 0.001). Additionally, forced expiratory volume in 1 second and the disease risk index were the components of the rPAM significantly associated with outcome. In our large real-world cohort with extended follow-up, the rPAM score was validated as an independent predictor of OS in patients with hematologic disorders undergoing allo-HCT.
移植前风险评分,如修订后的移植前死亡率评估(rPAM)评分,有助于预测接受异基因造血细胞移植(allo-HCT)患者的预后。由于rPAM尚未在患有不同疾病的异质性患者群体中进行外部验证,我们旨在在allo-HCT患者的真实世界队列中验证rPAM评分。共有429例患者纳入研究,他们在2008年至2015年期间接受了首次allo-HCT。评估了rPAM评分对allo-HCT后4年总生存(OS)、非复发死亡率(NRM)和复发累积发生率(CIR)的预测能力。此外,我们评估了rPAM评分对OS的影响,并使用单变量和多变量分析来确定OS的患者和移植相关预测因素。在rPAM评分<17、17-23、24-30和>30的类别中,4年时的OS概率分别为61%、36%、26%和10%,差异有统计学意义(<0.0001)。与CIR相反,rPAM评分较高的患者NRM显著增加(<0.001)。关于OS,4年时rPAM评分的受试者工作特征曲线下面积为0.676(95%置信区间[CI],0.625-0.727)。在多变量分析中,rPAM评分与OS相关,独立于预处理方案(每增加1个单位的调整后风险比,1.10;95%CI,1.06-1.10;<0.001)。此外,第1秒用力呼气量和疾病风险指数是与rPAM结果显著相关的组成部分。在我们这个具有延长随访时间的大型真实世界队列中,rPAM评分被验证为接受allo-HCT的血液系统疾病患者OS的独立预测因素。