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验证血液恶性肿瘤患者移植后生存预测的造血细胞移植综合风险(HCT-CR)模型。

Validation of a Hematopoietic Cell Transplant-Composite Risk (HCT-CR) Model for Post-Transplant Survival Prediction in Patients with Hematologic Malignancies.

机构信息

Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, Texas.

Center of Excellence in Clinical Epidemiology, Faculty of Medicine, Thammasat University, Pathumthani, Thailand.

出版信息

Clin Cancer Res. 2020 May 15;26(10):2404-2410. doi: 10.1158/1078-0432.CCR-19-3919. Epub 2020 Feb 4.

DOI:10.1158/1078-0432.CCR-19-3919
PMID:32019857
Abstract

PURPOSE

Allogeneic hematopoietic stem cell transplantation (AHCT) outcomes depend on disease and patient characteristics. We previously developed a novel prognostic model, hematopoietic cell transplant composite-risk (HCT-CR) by incorporating the refined disease risk index (DRI-R) and hematopoietic cell transplant-comorbidity/age index (HCT-CI/Age) to predict post-transplant survival in patients with acute myeloid leukemia and myelodysplastic syndrome. Here we aimed to validate and prove the generalizability of the HCT-CR model in an independent cohort of patients with hematologic malignancies receiving AHCT.

EXPERIMENTAL DESIGN

Data of consecutive adult patients receiving AHCT for various hematologic malignancies were analyzed. Patients were stratified into four HCT-CR risk groups. The discrimination, calibration performance, and clinical net benefit of the HCT-CR model were tested.

RESULTS

The HCT-CR model stratified patients into four risk groups with significantly different overall survival (OS). Three-year OS was 67.4%, 50%, 37.5%, and 29.9% for low, intermediate, high, and very high-risk group, respectively ( < 0.001). The HCT-CR model had better discrimination on OS prediction when compared with the DRI-R and HCT-CI/Age (C-index was 0.69 vs. 0.59 and 0.56, respectively, < 0.001). The decision curve analysis showed that HCT-CR model provided better clinical utility for patient selection for post-transplant clinical trial than the "treat all" or "treat none" strategy and the use of the DRI-R and HCT-CI/Age model separately.

CONCLUSIONS

The HCT-CR can be effectively used to predict post-transplant survival in patients with various hematologic malignancies. This composite model can identify patients who will benefit the most from transplantation and helps physicians in making decisions regarding post-transplant therapy to improve outcomes.

摘要

目的

异基因造血干细胞移植(allo-HSCT)的结果取决于疾病和患者特征。我们之前通过纳入改良疾病风险指数(DRI-R)和造血细胞移植合并症/年龄指数(HCT-CI/Age),开发了一种新的预后模型——造血细胞移植复合风险(HCT-CR),以预测急性髓系白血病和骨髓增生异常综合征患者移植后的生存情况。在此,我们旨在验证和证明该 HCT-CR 模型在接受 allo-HSCT 的血液系统恶性肿瘤患者的独立队列中的通用性。

实验设计

分析了连续接受 allo-HSCT 治疗的各种血液系统恶性肿瘤的成年患者的数据。患者被分为四个 HCT-CR 风险组。测试了 HCT-CR 模型的区分度、校准性能和临床净获益。

结果

HCT-CR 模型将患者分为四个具有显著不同总体生存(OS)的风险组。低、中、高和极高风险组的 3 年 OS 分别为 67.4%、50%、37.5%和 29.9%(<0.001)。与 DRI-R 和 HCT-CI/Age 相比,HCT-CR 模型在 OS 预测方面具有更好的区分度(C 指数分别为 0.69、0.59 和 0.56,均<0.001)。决策曲线分析表明,HCT-CR 模型在为移植后临床试验选择患者方面提供了比“治疗所有”或“不治疗任何”策略以及单独使用 DRI-R 和 HCT-CI/Age 模型更好的临床效用。

结论

HCT-CR 可有效用于预测各种血液系统恶性肿瘤患者移植后的生存情况。该复合模型可以识别最有可能从移植中受益的患者,并帮助医生在制定移植后治疗决策以改善结局方面做出决策。

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