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Scientists often need to analyze the samples in a study that responded to treatment in order to refine their hypotheses and find potential causal drivers of response. Natural variation in outcomes makes teasing apart responders from…

统计方法学 · 统计学 2025-02-14 Christopher Tosh , Boyuan Zhang , Wesley Tansey

Driven by the critical needs of biomanufacturing 4.0, we introduce a probabilistic knowledge graph hybrid model characterizing the risk- and science-based understanding of bioprocess mechanisms. It can faithfully capture the important…

机器学习 · 统计学 2022-10-03 Wei Xie , Keqi Wang , Hua Zheng , Ben Feng

From personalised medicine to targeted advertising, it is an inherent task to provide a sequence of decisions with historical covariates and outcome data. This requires understanding of both the dynamics and heterogeneity of treatment…

统计方法学 · 统计学 2022-06-22 Oscar Hernan Madrid Padilla , Yi Yu

Clinical trials are an integral component of medical research. Trials require careful design to, for example, maintain the safety of participants, use resources efficiently and allow clinically meaningful conclusions to be drawn. Adaptive…

统计方法学 · 统计学 2025-07-17 James M. McGree , Antony M. Overstall , Mark Jones , Robert K. Mahar

Estimating the impact of trauma treatment protocols is complicated by the high dimensional yet finite sample nature of trauma data collected from observational studies. Viscoelastic assays are highly predictive measures of hemostasis.…

应用统计 · 统计学 2022-09-27 Linqing Wei , Lucy Z. Kornblith , Alan Hubbard

Joint models (JMs) for longitudinal and time-to-event data are an important class of biostatistical models in health and medical research. When the study population consists of heterogeneous subgroups, the standard JM may be inadequate and…

统计方法学 · 统计学 2024-10-31 Sida Chen , Danilo Alvares , Marco Palma , Jessica K. Barrett

The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

应用统计 · 统计学 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

Network meta-analysis (NMA) is widely used in healthcare decision-making, where estimates of the effect of multiple treatments on outcomes are required. For time-to-event outcomes such as survival or disease progression the most common…

统计方法学 · 统计学 2025-09-15 David M. Phillippo , Ayman Sadek , Hugo Pedder , Nicky J. Welton

Joint models have proven to be an effective approach for uncovering potentially hidden connections between various types of outcomes, mainly continuous, time-to-event, and binary. Typically, longitudinal continuous outcomes are…

We consider the problem of partial identification, the estimation of bounds on the treatment effects from observational data. Although studied using discrete treatment variables or in specific causal graphs (e.g., instrumental variables),…

机器学习 · 计算机科学 2022-10-18 Vahid Balazadeh , Vasilis Syrgkanis , Rahul G. Krishnan

Adoptive Cell Transfer therapy of cancer is currently in full development and mathematical modeling is playing a critical role in this area. We study a stochastic model developed by Baar et al. in 2015 for modeling immunotherapy against…

种群与进化 · 定量生物学 2020-03-09 Modibo Diabate , Loren Coquille , Adeline Samson

Predicting the survival time of a cancer patient based on his/her genome-wide gene expression remains a challenging problem. For certain types of cancer, the effects of gene expression on survival are both weak and abundant, so identifying…

统计方法学 · 统计学 2018-09-03 Aaron J. Molstad , Li Hsu , Wei Sun

One of the barriers to the development of effective adoptive cell transfer therapies (ACT), specifically for genetically engineered T-cell receptors (TCRs), and chimeric antigen receptor (CAR) T-cells, is target antigen heterogeneity. It is…

We demonstrate a comprehensive semiparametric approach to causal mediation analysis, addressing the complexities inherent in settings with longitudinal and continuous treatments, confounders, and mediators. Our methodology utilizes a…

As standards of care advance, patients are living longer and once-fatal diseases are becoming manageable. Clinical trials increasingly focus on reducing disease burden, which can be quantified by the timing and occurrence of multiple…

Sepsis is a deadly condition affecting many patients in the hospital. Recent studies have shown that patients diagnosed with sepsis have significant mortality and morbidity, resulting from the body's dysfunctional host response to…

机器学习 · 计算机科学 2022-12-14 Ronald Moore , Rishikesan Kamaleswaran

Breast cancer patients may experience relapse or death after surgery during the follow-up period, leading to dependent censoring of relapse. This phenomenon, known as semi-competing risk, imposes challenges in analyzing treatment effects on…

统计方法学 · 统计学 2024-07-03 Tonghui Yu , Mengjiao Peng , Yifan Cui , Elynn Chen , Chixiang Chen

Penetrance, which plays a key role in genetic research, is defined as the proportion of individuals with the genetic variants (i.e., {genotype}) that cause a particular trait and who have clinical symptoms of the trait (i.e., {phenotype}).…

应用统计 · 统计学 2018-05-04 Seung Jun Shin , Ying Yuan , Louise C. Strong , Jasmina Bojadzieva , Wenyi Wang

Interval censoring occurs when event times are only known to fall between scheduled assessments, a common design in clinical trials, epidemiology, and reliability studies. Standard right-censoring methods, such as Kaplan-Meier and Cox…

统计方法学 · 统计学 2025-09-04 J. T. Korley

Dynamic treatment regimes are of growing interest across the clinical sciences as these regimes provide one way to operationalize and thus inform sequential personalized clinical decision making. A dynamic treatment regime is a sequence of…

统计方法学 · 统计学 2013-11-27 Eric B. Laber , Min Qian , Dan J. Lizotte , William E. Pelham , Susan A. Murphy