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Recently, phase II trials with multiple schedules (frequency of administrations) have become more popular, for instance in the development of treatments for atopic dermatitis. If the relationship of the dose and response is described by a…

Applications · Statistics 2020-11-17 Burak Kürsad Günhan , Paul Meyvisch , Tim Friede

Over the past decade, several targeted therapies (e.g. imatinib, dasatinib, nilotinib) have been developed to treat Chronic Myeloid Leukemia (CML). Despite an initial response to therapy, drug resistance remains a problem for some CML…

Tissues and Organs · Quantitative Biology 2017-02-08 Qie He , Junfeng Zhu , David Dingli , Jasmine Foo , Kevin Leder

eHealth technologies have been increasingly used to foster proactive self-management skills for patients with chronic diseases. However, it is challenging to provide each user with their desired support due to the dynamic and diverse nature…

Human-Computer Interaction · Computer Science 2023-12-20 Wei Wang , Hourieh Khalajzadeh , Anuradha Madugalla , Jennifer Mcintosh , Humphrey Obie

This paper develops a nonparametric model that represents how sequences of outcomes and treatment choices influence one another in a dynamic manner. In this setting, we are interested in identifying the average outcome for individuals in…

Econometrics · Economics 2019-01-16 Sukjin Han

The coronavirus disease 2019 (COVID-19) pandemic has exerted a profound impact on patients with end-stage renal disease relying on kidney dialysis to sustain their lives. Motivated by a request by the U.S. Centers for Medicare & Medicaid…

Methodology · Statistics 2022-09-02 Wenbo Wu , John D. Kalbfleisch , Jeremy M. G. Taylor , Jian Kang , Kevin He

We consider the problem of testing for treatment effect heterogeneity in observational studies, and propose a nonparametric test based on multisample U-statistics. To account for potential confounders, we use reweighted data where the…

Methodology · Statistics 2021-03-30 Maozhu Dai , Weining Shen , Hal S. Stern

Estimation of heterogeneous treatment effects is an active area of research. Most of the existing methods, however, focus on estimating the conditional average treatment effects of a single, binary treatment given a set of pre-treatment…

Methodology · Statistics 2025-05-30 Max Goplerud , Kosuke Imai , Nicole E. Pashley

To better understand effects of exposure to food allergens, food challenge studies are designed to slowly increase the dose of an allergen delivered to allergic individuals until an objective reaction occurs. These dose-to-failure studies…

Applications · Statistics 2019-08-30 Matthew W. Wheeler , Joost Westerhout , Joe L. Baumert , Benjamin C. Remington

End-stage chronic kidney disease (CKD) patients are facing a 30% rise for the risk of lethal cardiac events (LCE) compared to non-CKD patients. At the same time, these patients undergoing dialysis experience shifts in the potassium…

Signal Processing · Electrical Eng. & Systems 2020-06-11 Nicolas Pilia , Cristiana Corsi , Stefano Severi , Olaf Dössel , Axel Loewe

Purpose: Pulmonary embolism (PE) is a significant cause of mortality in the United States. The objective of this study is to implement deep learning (DL) models using Computed Tomography Pulmonary Angiography (CTPA), clinical data, and PE…

We investigate how to exploit structural similarities of an individual's potential outcomes (POs) under different treatments to obtain better estimates of conditional average treatment effects in finite samples. Especially when it is…

Machine Learning · Statistics 2021-10-26 Alicia Curth , Mihaela van der Schaar

This paper considers the identification of dynamic treatment effects with panel data, in complex designs where the treatment may not be binary and may not be absorbing. We first show that under no-anticipation and parallel-trends…

Econometrics · Economics 2025-12-23 Clément de Chaisemartin , Xavier D'Haultfœuille

Using state-level opioid overdose mortality data from 1999-2016, we simulated four time-varying treatment scenarios, which correspond to real-world policy dynamics (ramp up, ramp down, temporary and inconsistent). We then evaluated seven…

In the dynamic hospital setting, decision support can be a valuable tool for improving patient outcomes. Data-driven inference of future outcomes is challenging in this dynamic setting, where long sequences such as laboratory tests and…

Quantitative Methods · Quantitative Biology 2024-04-25 Alan D. Kaplan , Priyadip Ray , John D. Greene , Vincent X. Liu

We propose a dynamic allocation procedure that increases power and efficiency when measuring an average treatment effect in sequential randomized trials. Subjects arrive iteratively and are either randomized or paired via a matching…

Methodology · Statistics 2013-05-23 Adam Kapelner , Abba Krieger

Introduction: Chronic pain patients are at increased risk of opioid-misuse. Less is known about the unique risk conferred by each pain-management treatment, as treatments are typically implemented together, confounding their independent…

Patients with epilepsy can manifest short, sub-clinical epileptic "bursts" in addition to full-blown clinical seizures. We believe the relationship between these two classes of events---something not previously studied…

Machine Learning · Statistics 2014-07-29 Drausin F. Wulsin , Emily B. Fox , Brian Litt

Random effects meta-analysis is widely used for synthesizing studies under the assumption that underlying effects come from a normal distribution. However, under certain conditions the use of alternative distributions might be more…

Treatment-related lymphopenia is a frequent and clinically significant consequence of cancer therapy that can compromise immune-mediated tumor control and worsen patient outcomes. Despite its importance, no mechanistic framework exists to…

Medical Physics · Physics 2026-05-04 Vladislav Sandul , Marco Durante , Thomas Friedrich

A treatment regime is a deterministic function that dictates personalized treatment based on patients' individual prognostic information. There is a fast-growing interest in finding optimal treatment regimes to maximize expected long-term…

Statistics Theory · Mathematics 2016-11-25 Runchao Jiang , Wenbin Lu , Rui Song , Marie Davidian