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修订的移植前死亡率评分在接受异基因造血干细胞移植的急性髓系白血病患者中的验证。

Validation of the Revised Pretransplant Assessment of Mortality Score in Patients with Acute Myelogenous Leukemia Undergoing Allogeneic Hematopoietic Stem Cell Transplantation.

机构信息

Universitätsklinikum Carl Gustav Carus der TU Dresden, Medizinische Klinik und Poliklinik I, Dresden, Germany.

Universitätsklinikum Carl Gustav Carus der TU Dresden, Medizinische Klinik und Poliklinik I, Dresden, Germany.

出版信息

Biol Blood Marrow Transplant. 2018 Sep;24(9):1947-1951. doi: 10.1016/j.bbmt.2018.05.021. Epub 2018 May 21.

Abstract

Despite recent advances, allogeneic hematopoietic stem cell transplantation (allo-HSCT) continues to be accompanied by a high rate of morbidity and mortality. Several scores have been developed to predict outcome after allo-HSCT. The recently revised Pretransplant Assessment of Mortality (PAM) score is based on patient age, donor type, disease risk, cytomegalovirus (CMV) serostatus of patient and donor, and forced expiratory volume in 1 second (FEV). The aim of this study was to analyze the predictive power of the PAM score in an independent large cohort of patients with acute myelogenous leukemia (AML). We selected adult patients with AML who underwent a first allo-HSCT at the University Hospital of Dresden, a tertiary care hospital with a large transplantation program. All adult patients treated between January 1, 2003, and July 1, 2015, were included. The PAM score was calculated as described previously. Overall survival (OS), cumulative incidence of relapse (CIR), and nonrelapse mortality (NRM) after allo-HSCT were analyzed. Age, AML type, sex match, CMV match, donor type, European Leukemia Net risk classification, type of conditioning, disease stage, and PAM score as a continuous variable were selected a priori for multivariate Cox regression analyses. A total of 544 patients met the inclusion criteria. The median patient age was 57 years. With a median follow-up of 47 months (range, 1 to 161 months), the estimated OS for the whole cohort at 4 years was 43%, with a CIR of 30% and an NRM of 31%. The probability of OS at 4 years was 65% for patients with a PAM score of 0, 52% in those with a PAM score of 1, 33% in those with a PAM score of 2, and 22% in those with a PAM score of 3 (P < .001, log-rank test). Both the CIR and NRM increased with higher PAM scores (P = .005 and P < .001, respectively, Gray test). In multivariate analysis, age (hazard ratio [HR], 1.02 per year; P = .004), disease stage (primary induction failure versus first complete remission (CR1); HR, 1.5; P = .03), and the PAM score (HR 1.04; P = .03) had a significant impact on OS. This is the first independent validation of the revised PAM score allowing for simple and valid estimation of transplantation outcomes. It can serve as an important tool in counseling patients with AML, as well as in designing future trials.

摘要

尽管最近取得了进展,但异基因造血干细胞移植(allo-HSCT)仍然伴随着高发病率和死亡率。已经开发了几种评分来预测 allo-HSCT 后的结果。最近修订的移植前死亡率(PAM)评分基于患者年龄、供体类型、疾病风险、患者和供体的巨细胞病毒(CMV)血清状态以及 1 秒用力呼气量(FEV)。本研究的目的是在急性髓细胞白血病(AML)的独立大队列患者中分析 PAM 评分的预测能力。我们选择了在三级护理医院、大型移植项目的德累斯顿大学医院接受首次 allo-HSCT 的成年 AML 患者。纳入了 2003 年 1 月 1 日至 2015 年 7 月 1 日期间接受治疗的所有成年患者。按照先前的描述计算 PAM 评分。分析 allo-HSCT 后的总生存率(OS)、累积复发率(CIR)和非复发死亡率(NRM)。年龄、AML 类型、性别匹配、CMV 匹配、供体类型、欧洲白血病网风险分类、预处理类型、疾病分期和 PAM 评分作为连续变量,用于多变量 Cox 回归分析。共有 544 名患者符合纳入标准。患者的中位年龄为 57 岁。中位随访 47 个月(范围 1 至 161 个月),整个队列 4 年的 OS 估计值为 43%,CIR 为 30%,NRM 为 31%。PAM 评分为 0 的患者 4 年 OS 概率为 65%,PAM 评分为 1 的患者为 52%,PAM 评分为 2 的患者为 33%,PAM 评分为 3 的患者为 22%(P<.001,对数秩检验)。随着 PAM 评分的升高,OS(P=.005 和 P<.001,灰色检验)和 NRM(P=.005 和 P<.001,灰色检验)均增加。多变量分析显示,年龄(每增加 1 年,风险比[HR]为 1.02;P=.004)、疾病分期(原发诱导失败与首次完全缓解[CR1];HR,1.5;P=.03)和 PAM 评分(HR 1.04;P=.03)对 OS 有显著影响。这是对修订后的 PAM 评分的首次独立验证,可简单有效地评估移植结果。它可以作为 AML 患者咨询的重要工具,也可以作为未来试验的设计工具。

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