Liang Muxuan, Zhao Yingqi, Lin Daniel W, Cooperberg Matthew, Zheng Yingye
Department of Biostatistics, University of Florida, Gainesville, FL 32611, United States.
Public Health Sciences Division, Fred Hutchinson Cancer Center, Seattle, WA 98109, United States.
Biometrics. 2025 Apr 2;81(2). doi: 10.1093/biomtc/ujaf067.
Active surveillance (AS) using repeated biopsies to monitor disease progression has been a popular alternative to immediate surgical intervention in cancer care. However, a biopsy procedure is invasive and sometimes leads to severe side effects of infection and bleeding. To reduce the burden of repeated surveillance biopsies, biomarker-assistant decision rules are sought to replace the fix-for-all regimen with tailored biopsy intensity for individual patients. Constructing or evaluating such decision rules is challenging. The key AS outcome is often ascertained subject to interval censoring. Furthermore, patients will discontinue participation in the AS study once they receive a positive surveillance biopsy. Thus, patient dropout is affected by the outcomes of these biopsies. This work proposes a non-parametric kernel-based method to estimate a tailored AS strategy's true positive rates (TPRs) and true negative rates (TNRs), accounting for interval censoring and immediate dropouts. We develop a weighted classification framework based on these estimates to estimate the optimally tailored AS strategy and further incorporate the cost-benefit ratio for cost-effectiveness in medical decision-making. Theoretically, we provide a uniform generalization error bound of the derived AS strategy, accommodating all possible trade-offs between TPRs and TNRs. Simulation and application to a prostate cancer surveillance study show the superiority of the proposed method.
在癌症治疗中,使用重复活检来监测疾病进展的主动监测(AS)已成为立即进行手术干预的一种流行替代方案。然而,活检程序具有侵入性,有时会导致感染和出血等严重副作用。为了减轻重复监测活检的负担,人们寻求生物标志物辅助决策规则,以针对个体患者采用量身定制的活检强度来取代一刀切的方案。构建或评估此类决策规则具有挑战性。主动监测的关键结果通常受到区间删失的影响。此外,一旦患者的监测活检结果呈阳性,他们将停止参与主动监测研究。因此,患者退出受这些活检结果的影响。这项工作提出了一种基于非参数核的方法,用于估计量身定制的主动监测策略的真阳性率(TPR)和真阴性率(TNR),同时考虑区间删失和立即退出的情况。我们基于这些估计值开发了一个加权分类框架,以估计最优的量身定制的主动监测策略,并在医疗决策中进一步纳入成本效益比以实现成本效益。从理论上讲,我们为推导得出的主动监测策略提供了一个统一的泛化误差界,涵盖了真阳性率和真阴性率之间所有可能的权衡。对前列腺癌监测研究的模拟和应用表明了所提方法的优越性。