Validation and refinement of the Disease Risk Index for allogeneic stem cell transplantation
Validation and refinement of the Disease Risk Index for allogeneic stem cell transplantation
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DOI：
10.1182/blood-2014-01-552984
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发表时间：
2014-06-05
期刊：
影响因子：
20.3
通讯作者：
Saber, Wael
中科院分区：
文献类型：
--
作者：
Armand, Philippe;Kim, Haesook T.;Saber, Wael
由于异基因造血细胞移植(HCT)的结果主要受疾病类型和状态的影响，因此能够根据疾病风险对HCT患者进行分层是至关重要的。疾病风险指数(DRI)就是为此目的而开发的。在这项研究中，我们分析了2008年至2010年期间向国际血液和骨髓移植研究中心报告的13131名接受HCT的患者。DRI将患者分成4组，两年总生存率(OS)从%到24%不等，是最强的预后因素，无论年龄、预适应强度、移植物来源或供者类型。使用随机选择的9849名患者的训练组来改进DRI，使用OS的多变量回归模型。与原始DRI或其他现有方案相比，改进的DRI对其余3282名患者的预测能力有所提高。这一经过验证和改进的DRI可用作4组或3组指数，具体取决于研究中队列的大小，用于预测；促进单中心、多中心或注册研究的解释；调整中心结果数据；以及对进入临床试验的患者进行分层，以招募不同疾病类别的患者。
Because the outcome of allogeneic hematopoietic cell transplantation (HCT) is predominantly influenced by disease type and status, it is essential to be able to stratify patients undergoing HCT by disease risk. The Disease Risk Index (DRI) was developed for this purpose. In this study, we analyzed 13 131 patients reported to the Center for International Blood and Marrow Transplant Research who underwent HCT between 2008 and 2010. TheDRI stratified patients into 4 groups with 2-year overall survival (OS) ranging from 64% to 24% and was the strongest prognostic factor, regardless of age, conditioning intensity, graft source, or donor type. A randomly selected training subgroup of 9849 patients was used to refine the DRI, using a multivariable regression model for OS. This refined DRI had improved prediction ability for the remaining 3282 patients compared with the original DRI or other existing schemes. This validated and refined DRI can be used as a 4- or 3-group index, depending on the size of the cohort under study, for prognostication; to facilitate the interpretation of single-center, multicenter, or registry studies; to adjust center outcome data; and to stratify patients entering clinical trials that enroll patients across disease categories.