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Survival analysis is a challenging variation of regression modeling because of the presence of censoring, where the outcome measurement is only partially known, due to, for example, loss to follow up. Such problems come up frequently in…

机器学习 · 计算机科学 2022-06-28 Chirag Nagpal , Steve Yadlowsky , Negar Rostamzadeh , Katherine Heller

An important task in clinical medicine is the construction of risk prediction models for specific subgroups of patients based on high-dimensional molecular measurements such as gene expression data. Major objectives in modeling…

统计方法学 · 统计学 2020-03-23 Katrin Madjar , Jörg Rahnenführer

In survival analysis it often happens that some subjects under study do not experience the event of interest; they are considered to be `cured'. The population is thus a mixture of two subpopulations: the one of cured subjects, and the one…

统计理论 · 数学 2017-01-16 Valentin Patilea , Ingrid Van Keilegom

Progress in immunotherapy revolutionized the treatment landscape for advanced lung cancer, raising survival expectations beyond those that were historically anticipated with this disease. In the present study, we describe the methods for…

应用统计 · 统计学 2019-11-25 Lizet Sanchez , Patricia Lorenzo-Luaces , Claudia Fonte , Agustin Lage

One of the most common ways researchers compare survival outcomes across treatments when confounding is present is using Cox regression. This model is limited by its underlying assumption of proportional hazards; in some cases, substantial…

应用统计 · 统计学 2021-02-02 Elizabeth A. Handorf , Marc Smaldone , Sujana Movva , Nandita Mitra

Clinical trials with time-to-event endpoints, such as overall survival (OS) or progression-free survival (PFS), are fundamental for evaluating new treatments, particularly in immuno-oncology. However, modern therapies, such as…

统计方法学 · 统计学 2025-09-10 James Salsbury , Jeremy Oakley , Steven Julious , Lisa Hampson

In this study, we address the challenge of survival analysis within heterogeneous patient populations, where traditional reliance on a single regression model such as the Cox proportional hazards (Cox PH) model often falls short.…

统计方法学 · 统计学 2025-04-15 Ahmad Talafha

Analysing subgroups defined by biomarkers is of increasing importance in clinical research. In some situations the biomarker is subject to misclassification error, meaning the true subgroups are identified with imperfect sensitivity and…

统计方法学 · 统计学 2018-01-16 Fang Wan , Andrew C. Titman , Thomas F. Jaki

In this paper, a methodology is proposed that enables to analyze the sensitivity of the outcome of a therapy to unavoidable high dispersion of the patient specific parameters on one hand and to the choice of the parameters that define the…

系统与控制 · 电气工程与系统科学 2022-05-17 Mazen Alamir

Truncation is a statistical phenomenon that occurs in many time to event studies. For example, autopsy-confirmed studies of neurodegenerative diseases are subject to an inherent left and right truncation, also known as double truncation.…

统计方法学 · 统计学 2018-03-28 Lior Rennert , Sharon X. Xie

In observational studies with survival or time-to-event outcomes, a propensity score weighted marginal Cox proportional hazard model with the treatment variable as the only predictor is commonly used to estimate the causal marginal hazard…

统计方法学 · 统计学 2026-02-02 Zixian Zhao , Chengxin Yang , Fan Li

Chemotherapy is one of the primary modalities of cancer treatment. Chemotherapy drug administration is a complex problem that often requires expensive clinical trials to evaluate potential regimens. One way to alleviate this burden and…

While the use of combination therapy is increasing in prevalence for cancer treatment, it is often difficult to predict the exact interactions between different treatment forms, and their synergistic/antagonistic effects on patient health…

最优化与控制 · 数学 2021-01-29 Jayanth Pratap

With medical tests becoming increasingly available, concerns about over-testing and over-treatment dramatically increase. Hence, it is important to understand the influence of testing on treatment selection in general practice. Most…

统计方法学 · 统计学 2020-08-11 Yun Li , Irina Bondarenko , Michael R. Elliott , Timothy P. Hofer , Jeremy M. G. Taylor

The treatment effects of the same therapy observed from multiple clinical trials can often be very different. Yet the patient characteristics accounting for these differences may not be identifiable in real world practice. There needs to be…

统计理论 · 数学 2015-03-20 Tinghui Yu , Yabing Mai , Sherry Liu , Xiaofei Hu

The Cox regression, a semi-parametric method of survival analysis, is extremely popular in biomedical applications. The proportional hazards assumption is a key requirement in the Cox model. To accommodate non-proportional hazards, we…

统计方法学 · 统计学 2022-06-13 Alexander Begun , Elena Kulinskaya

This paper addresses patient heterogeneity associated with prediction problems in biomedical applications. We propose a systematic hypothesis testing approach to determine the existence of patient subgroup structure and the number of…

统计方法学 · 统计学 2021-01-08 Xu Gao , Weining Shen , Jing Ning , Ziding Feng , Jianhua Hu

Cell heterogeneity plays an important role in patient responses to drug treatments. In many cancers, it is associated with poor treatment outcomes. Many modern drug combination therapies aim to exploit cell heterogeneity, but determining…

Significant evidence has become available that emphasizes the importance of personalization in medicine. In fact, it has become a common belief that personalized medicine is the future of medicine. The core of personalized medicine is the…

统计方法学 · 统计学 2020-04-30 Qiong Zhang , Amin Khademi , Yongjia Song

We explore whether survival model performance in underrepresented high- and low-risk subgroups - regions of the prognostic spectrum where clinical decisions are most consequential - can be improved through targeted restructuring of the…

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