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治疗相关性与初发性骨髓增生异常综合征患者风险分层工具的临床结局和预后预测价值比较:代表 MDS 临床研究联盟的报告。

Comparison of clinical outcomes and prognostic utility of risk stratification tools in patients with therapy-related vs de novo myelodysplastic syndromes: a report on behalf of the MDS Clinical Research Consortium.

机构信息

Section of Hematology, Department of Internal Medicine, Yale University, and Yale Comprehensive Cancer Center, New Haven, CT, USA.

Department of Malignant Hematology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL, USA.

出版信息

Leukemia. 2017 Jun;31(6):1391-1397. doi: 10.1038/leu.2017.33. Epub 2017 Jan 23.

DOI:10.1038/leu.2017.33
PMID:28111463
Abstract

While therapy-related (t)-myelodysplastic syndromes (MDS) have worse outcomes than de novo MDS (d-MDS), some t-MDS patients have an indolent course. Most MDS prognostic models excluded t-MDS patients during development. The performances of the International Prognostic Scoring System (IPSS), revised IPSS (IPSS-R), MD Anderson Global Prognostic System (MPSS), WHO Prognostic Scoring System (WPSS) and t-MDS Prognostic System (TPSS) were compared among patients with t-MDS. Akaike information criteria (AIC) assessed the relative goodness of fit of the models. We identified 370 t-MDS patients (19%) among 1950 MDS patients. Prior therapy included chemotherapy alone (48%), chemoradiation (31%), and radiation alone in 21%. Median survival for t-MDS patients was significantly shorter than for d-MDS (19 vs 46 months, P<0.005). All models discriminated survival in t-MDS (P<0.005 for each model). Patients with t-MDS had a significantly higher hazard of death relative to d-MDS in every risk model, and had inferior survival compared to patients with d-MDS within all risk group categories. AIC Scores (lower is better) were 2316 (MPSS), 2343 (TPSS), 2343 (IPSS-R), 2361 (WPSS) and 2364 (IPSS). In conclusion, subsets of t-MDS patients with varying clinical outcomes can be identified using conventional risk stratification models. The MPSS, TPSS and IPSS-R provide the best predictive power.

摘要

虽然治疗相关(t)-骨髓增生异常综合征(MDS)的预后比初发 MDS(d-MDS)差,但有些 t-MDS 患者的病程呈惰性。大多数 MDS 预后模型在开发过程中排除了 t-MDS 患者。比较了 t-MDS 患者中国际预后评分系统(IPSS)、修订的 IPSS(IPSS-R)、MD 安德森全球预后系统(MPSS)、世界卫生组织预后评分系统(WPSS)和 t-MDS 预后系统(TPSS)的性能。Akaike 信息准则(AIC)评估了模型的相对拟合优度。我们在 1950 例 MDS 患者中发现了 370 例 t-MDS 患者(19%)。既往治疗包括单纯化疗(48%)、放化疗(31%)和单纯放疗(21%)。t-MDS 患者的中位生存时间明显短于 d-MDS(19 个月 vs 46 个月,P<0.005)。所有模型均能区分 t-MDS 患者的生存情况(每种模型均 P<0.005)。与 d-MDS 相比,t-MDS 患者在每个风险模型中的死亡风险显著增加,并且在所有风险组类别中,t-MDS 患者的生存情况均劣于 d-MDS 患者。AIC 评分(越低越好)分别为 2316(MPSS)、2343(TPSS)、2343(IPSS-R)、2361(WPSS)和 2364(IPSS)。结论:使用传统风险分层模型可以识别出具有不同临床结局的 t-MDS 患者亚组。MPSS、TPSS 和 IPSS-R 提供了最佳的预测能力。

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