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利用真实世界数据开发和验证多发性骨髓瘤治疗线识别算法。

Development and validation of algorithms for identifying lines of therapy in multiple myeloma using real-world data.

机构信息

Division of Hematology/Oncology, Department of Medicine, Mayo Clinic, Jacksonville, FL 32224, USA.

Global Evidence & Outcomes, Takeda Development Center Americas, Inc. (TDCA), Lexington, MA 02421, USA.

出版信息

Future Oncol. 2024 May;20(15):981-995. doi: 10.2217/fon-2023-0696. Epub 2024 Jan 17.

Abstract

To validate algorithms based on electronic health data to identify composition of lines of therapy (LOT) in multiple myeloma (MM). This study used available electronic health data for selected adults within Henry Ford Health (Michigan, USA) newly diagnosed with MM in 2006-2017. Algorithm performance in this population was verified via chart review. As with prior oncology studies, good performance was defined as positive predictive value (PPV) ≥75%. Accuracy for identifying LOT1 (N = 133) was 85.0%. For the most frequent regimens, accuracy was 92.5-97.7%, PPV 80.6-93.8%, sensitivity 88.2-89.3% and specificity 94.3-99.1%. Algorithm performance decreased in subsequent LOTs, with decreasing sample sizes. Only 19.5% of patients received maintenance therapy during LOT1. Accuracy for identifying maintenance therapy was 85.7%; PPV for the most common maintenance therapy was 73.3%. Algorithms performed well in identifying LOT1 - especially more commonly used regimens - and slightly less well in identifying maintenance therapy therein.

摘要

验证基于电子健康数据的算法,以确定多发性骨髓瘤(MM)中的治疗线组成(LOT)。本研究使用了美国密歇根州亨利福特健康中心(Henry Ford Health) 2006 年至 2017 年间新诊断为 MM 的选定成年患者的现有电子健康数据。通过病历回顾验证了该人群中算法的性能。与之前的肿瘤学研究一样,良好的性能定义为阳性预测值(PPV)≥75%。识别 LOT1(N=133)的准确率为 85.0%。对于最常见的方案,准确率为 92.5-97.7%,PPV 为 80.6-93.8%,灵敏度为 88.2-89.3%,特异性为 94.3-99.1%。随着样本量的减少,后续 LOT 中的算法性能下降。只有 19.5%的患者在 LOT1 期间接受维持治疗。识别维持治疗的准确率为 85.7%;最常见维持治疗的 PPV 为 73.3%。该算法在识别 LOT1 方面表现良好-尤其是更常用的方案-,在识别其中的维持治疗方面表现稍差。

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