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We study the problem of sequential experimental design to estimate the parametric component of a partially linear model with a Gaussian process prior. We consider an active learning setting where an experimenter adaptively decides which…

Methodology · Statistics 2022-11-07 Shunsuke Horii

Acute lymphoblastic leukemia is the most common malignancy in childhood. Successful treatment requires initial high-intensity chemotherapy, followed by low-intensity oral maintenance therapy with oral 6-mercaptopurine (6MP) and methotrexate…

Quantitative Methods · Quantitative Biology 2020-01-06 Felix Jost , Jakob Zierk , Thuy T. T. Le , Thomas Raupach , Manfred Rauh , Meinolf Suttorp , Martin Stanulla , Markus Metzler , Sebastian Sager

A dynamic treatment regime is a sequence of decision rules in which each decision rule recommends treatment based on features of patient medical history such as past treatments and outcomes. Existing methods for estimating optimal dynamic…

Methodology · Statistics 2015-05-22 Kristin A. Linn , Eric B. Laber , Leonard A. Stefanski

Mendelian diseases are determined by a single mutation in a given gene. However, in the case of diseases with late onset, the age at onset is variable; it can even be the case that the onset is not observed in a lifetime. Estimating the…

Applications · Statistics 2016-07-15 Flora Alarcon , Gregory Nuel , Violaine Plante-Bordeneuve

Accurate models of clinical actions and their impacts on disease progression are critical for estimating personalized optimal dynamic treatment regimes (DTRs) in medical/health research, especially in managing chronic conditions.…

Methodology · Statistics 2021-02-19 William Hua , Hongyuan Mei , Sarah Zohar , Magali Giral , Yanxun Xu

Methods for estimating heterogeneous treatment effect in observational data have largely focused on continuous or binary outcomes, and have been relatively less vetted with survival outcomes. Using flexible machine learning methods in the…

Applications · Statistics 2021-07-09 Liangyuan Hu , Jiayi Ji , Fan Li

For many infectious disease outbreaks, the at-risk population changes their behavior in response to the outbreak severity, causing the transmission dynamics to change in real-time. Behavioral change is often ignored in epidemic modeling…

Methodology · Statistics 2023-10-25 Caitlin Ward , Rob Deardon , Alexandra M. Schmidt

Acute lymphoblastic leukemia (ALL) is a heterogeneous hematologic malignancy involving the abnormal proliferation of immature lymphocytes, accounting for most pediatric cancer cases. ALL management in children has seen great improvement in…

In semicompeting risks problems, nonterminal time-to-event outcomes such as time to hospital readmission are subject to truncation by death. These settings are often modeled with illness-death models for the hazards of the terminal and…

Methodology · Statistics 2019-02-27 Leah Comment , Fabrizia Mealli , Sebastien Haneuse , Corwin Zigler

In recent years, research interest in personalised treatments has been growing. However, treatment effect heterogeneity and possibly time-varying treatment effects are still often overlooked in clinical studies. Statistical tools are needed…

Methodology · Statistics 2023-10-27 Caterina Gregorio , Giovanni Baj , Giulia Barbati , Francesca Ieva

In randomized trials and observational studies, it is often necessary to evaluate the extent to which an intervention affects a time-to-event outcome, which is only partially observed due to right censoring. For instance, in infectious…

Methodology · Statistics 2024-12-16 Yutong Jin , Peter B. Gilbert , Aaron Hudson

For a trial with primary endpoint overall survival for a molecule with curative potential, statistical methods that rely on the proportional hazards assumption may underestimate the power and the time to final analysis. We show how a cure…

Applications · Statistics 2023-04-17 Kaspar Rufibach , Dominik Heinzmann , Annabelle Monnet

Prognostication for lung cancer, a leading cause of mortality, remains a complex task, as it needs to quantify the associations of risk factors and health events spanning a patient's entire life. One challenge is that an individual's…

Machine Learning · Statistics 2025-08-28 Stephen Salerno , Yi Li

Dynamic models are widely used to mathematically describe biological phenomena that evolve over time. One important area of application is leukaemia research, where leukaemia cells are genetically modified in preclinical studies to explore…

Methodology · Statistics 2026-02-02 Julian Wäsche , Romina Ludwig , Irmela Jeremias , Christiane Fuchs

Causal machine learning (CML) enables individualized estimation of treatment effects, offering critical advantages over traditional correlation-based methods. However, existing approaches for medical survival data with censoring such as…

We present a three-stage probabilistic model for the progression of Chronic Myeloid Leukemia (CML), as manifested by the leukemic stem cells, progenitor cells and mature leukemic cells. This progression is captured through the process of…

Populations and Evolution · Quantitative Biology 2022-04-25 Sonjoy Pan , Siddhartha P. Chakrabarty , Soumyendu Raha

Robust decision making involves making decisions in the presence of uncertainty and is often used in critical domains such as healthcare, supply chains, and finance. Causality plays a crucial role in decision-making as it predicts the…

Methodology · Statistics 2025-07-23 Saideep Nannapaneni , Joseph Sakaya , Kyle Caron , Pedro HM Albuquerque , Zaid Tashman

In longitudinal observational studies with time-to-event outcomes, a common objective in causal analysis is to estimate the causal survival curve under hypothetical intervention scenarios. The g-formula is a useful tool for this analysis.…

Methodology · Statistics 2025-04-14 Xinyuan Chen , Liangyuan Hu , Fan Li

The choice of the most effective treatment may eventually be influenced by breast cancer survival prediction. To predict the chances of a patient surviving, a variety of techniques were employed, such as statistical, machine learning, and…

Machine Learning · Computer Science 2023-04-18 Khaoula Chtouki , Maryem Rhanoui , Mounia Mikram , Kamelia Amazian , Siham Yousfi

Random allocation models used in clinical trials aid researchers in determining which of a particular treatment provides the best results by reducing bias between groups. Often however, this determination leaves researchers battling ethical…

Applications · Statistics 2020-08-04 Albert. H. Lee , Edward L Boone , Roy T. Sabo , Erin Donahue