Dong Sharlene, Kansagra Ankit, Kaur Gurbakhash, Barcellos Anna, Belli Andrew J, Fernandes Laura L, Hansen Eric, Ambrose Jacob, Bai Claire, Zettler Christina M, He Ming, Wang Ching-Kun
Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX.
Harold C. Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center, Dallas, TX.
JCO Clin Cancer Inform. 2025 Apr;9:e2400233. doi: 10.1200/CCI-24-00233. Epub 2025 Apr 16.
Accurate survival data are critical for high-quality outcomes research. It has been documented that mortality data capture in the real-world setting may be prone to missingness. Our study sought to evaluate the validity of a composite real-world mortality variable compared with the National Death Index (NDI) as the gold standard.
This was a retrospective, observational research study of patients with hematologic malignancies treated in the United States. Adult patients diagnosed with one of the following cancers between January 1, 2012, and December 31, 2020, were included: AML, chronic lymphocytic leukemia, diffuse large B-cell lymphoma, follicular lymphoma, marginal zone lymphoma, multiple myeloma, and myelodysplastic syndrome. Validation metrics (sensitivity, specificity, positive predictive value [PPV], and negative predictive value [NPV]) and date concordance (exact, ±7, 15, and 30 days) were assessed.
The final study population included N = 21,565 patients across seven cancer types. Validation metrics showed high sensitivity (87.8%), specificity (95.7%), PPV (90.9%), and NPV (94.1%) when comparing the composite real-world mortality variable with the NDI. Exact date concordance was observed in 88.0% of patients, and concordance rates for 7-, 15-, and 30-day intervals were 93.1%, 93.8%, and 94.3%, respectively.
Our study found that a composite mortality variable leveraging multiple data sources yields high validity when compared against the gold-standard NDI. Given evidence highlighting the challenges of mortality data documentation in the real-world setting, the use of a composite mortality variable can provide significant benefits in quality of documentation and research results.
准确的生存数据对于高质量的结局研究至关重要。有文献记载，在现实环境中死亡率数据的收集可能容易出现缺失情况。我们的研究旨在评估与作为金标准的国家死亡指数（NDI）相比，一个综合的现实世界死亡率变量的有效性。
这是一项对在美国接受治疗的血液系统恶性肿瘤患者进行的回顾性观察研究。纳入了2012年1月1日至2020年12月31日期间被诊断患有以下癌症之一的成年患者：急性髓系白血病、慢性淋巴细胞白血病、弥漫性大B细胞淋巴瘤、滤泡性淋巴瘤、边缘区淋巴瘤、多发性骨髓瘤和骨髓增生异常综合征。评估了验证指标（敏感性、特异性、阳性预测值[PPV]和阴性预测值[NPV]）以及日期一致性（精确、±7天、15天和30天）。
最终研究人群包括来自七种癌症类型的N = 21,565名患者。将综合的现实世界死亡率变量与NDI进行比较时，验证指标显示出高敏感性（87.8%）、特异性（95.7%）、PPV（90.9%）和NPV（94.1%）。88.0%的患者观察到精确日期一致性，7天、15天和30天间隔的一致性率分别为93.1%、93.8%和94.3%。
我们的研究发现，与金标准NDI相比，利用多个数据源的综合死亡率变量具有较高的有效性。鉴于有证据突出了现实环境中死亡率数据记录的挑战，使用综合死亡率变量可以在记录质量和研究结果方面带来显著益处。
