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In the first stage of a two-stage study, the researcher uses a statistical model to impute the unobserved exposures. In the second stage, imputed exposures serve as covariates in epidemiological models. Imputation error in the first stage…

Applications · Statistics 2021-07-19 Ron Sarafian , Itai Kloog , Jonathan D. Rosenblatt

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…

Methodology · Statistics 2020-04-30 Qiong Zhang , Amin Khademi , Yongjia Song

Efficient adversarial attack methods are critical for assessing the robustness of computer vision models. In this paper, we reconstruct the optimization objective for generating adversarial examples as "maximizing the difference between the…

Computer Vision and Pattern Recognition · Computer Science 2025-09-03 Xinlei Liu , Tao Hu , Peng Yi , Weitao Han , Jichao Xie , Baolin Li

An unprecedented number of new cancer targets are in development, and most are being developed in combination therapies. Early oncology development is strategically challenged in choosing the best combinations to move forward to late stage…

Applications · Statistics 2021-01-08 Linda Z. Sun , Cai , Wu , Xiaoyun , Li , Cong Chen , Emmett V. Schmidt

An important objective in the development of targeted therapies is to identify the populations where the treatment under consideration has positive benefit risk balance. We consider pivotal clinical trials, where the efficacy of a treatment…

Phase I dose-finding studies aim at identifying the maximal tolerated dose (MTD). It is not uncommon that several dose-finding studies are conducted, although often with some variation in the administration mode or dose panel. For instance,…

Quantitative Methods · Quantitative Biology 2021-03-24 Moreno Ursino , Christian Röver , Sarah Zohar , Tim Friede

Molecular optimization is a crucial aspect of drug discovery, aimed at refining molecular structures to enhance drug efficacy and minimize side effects, ultimately accelerating the overall drug development process. Many molecular…

Biomolecules · Quantitative Biology 2025-05-15 Kun Li , Xiantao Cai , Jia Wu , Shirui Pan , Huiting Xu , Bo Du , Wenbin Hu

Purpose: We propose a general framework for quantifying predictive uncertainties of dose-related quantities and leveraging this information in a dose mimicking problem in the context of automated radiation therapy treatment planning.…

Medical Physics · Physics 2021-09-08 Tianfang Zhang , Rasmus Bokrantz , Jimmy Olsson

The study of combinations of drugs/drug-schedules gained increasing attention in various therapeutic areas recently. In oncology, the aim of phase I combination clinical trial is to find the maximum tolerated combination (MTC). Many…

Methodology · Statistics 2025-05-09 Weishi Chen , Pavel Mozgunov

Multi-arm multi-stage trial designs can bring notable gains in efficiency to the drug development process. However, for normally distributed endpoints, the determination of a design typically depends on the assumption that the patient…

Methodology · Statistics 2017-10-11 Michael Grayling , James Wason , Adrian Mander

When planning an oncology clinical trial, the usual approach is to assume proportional hazards and even an exponential distribution for time-to-event endpoints. Often, besides the gold-standard endpoint overall survival (OS),…

Applications · Statistics 2023-12-19 Alexandra Erdmann , Jan Beyersmann , Kaspar Rufibach

Stochastic equations play an important role in computational science, due to their ability to treat a wide variety of complex statistical problems. However, current algorithms are strongly limited by their sampling variance, which scales…

Numerical Analysis · Mathematics 2017-01-04 Bogdan Opanchuk , Simon Kiesewetter , Peter D. Drummond

We propose a novel Phase I intra-patient dose-escalation design tailored for multi-cycle immunotherapy settings, in which toxicity at a fixed dose level is clinically expected to decrease over successive treatment cycles. This design was…

This work considers the problem of personalized dose guidance using Bayesian optimization that learns the optimum drug dose tailored to each individual, thus improving therapeutic outcomes. Safe learning using interior point method ensures…

Optimization and Control · Mathematics 2022-11-01 Dinesh Krishnamoorthy , Francis J. Doyle

The primary objective of Phase I oncology trials is to assess the safety and tolerability of novel therapeutics. Conventional dose escalation methods identify the maximum tolerated dose (MTD) based on dose-limiting toxicity (DLT). However,…

Methodology · Statistics 2024-09-02 Yunlong Yang , Ying Yuan

Dose-finding studies are frequently conducted to evaluate the effect of different doses or concentration levels of a compound on a response of interest. Applications include the investigation of a new medicinal drug, a herbicide or…

Applications · Statistics 2011-08-01 Björn Bornkamp , Frank Bretz , Holger Dette , José Pinheiro

Because of the high cost of commercial genotyping chip technologies, many investigations have used a two-stage design for genome-wide association studies, using part of the sample for an initial discovery of ``promising'' SNPs at a less…

While chemoresistance in primary tumors is well-studied, much less is known about the influence of systemic chemotherapy on the development of drug resistance at metastatic sites. In this work, we use a hybrid spatial model of tumor…

Tissues and Organs · Quantitative Biology 2016-03-04 Ami B. Shah , Katarzyna A. Rejniak , Jana L. Gevertz

Immunotherapies and targeted therapies have gained popularity due to their promising therapeutic effects across multiple treatment areas. The focus of early phase dose-finding clinical trials has shifted from finding the maximum tolerated…

Methodology · Statistics 2023-12-27 Hao Sun , Jieqi Tu

Many phase II clinical trials have used survival outcomes as the primary endpoints in recent decades. Suppose the radiotherapy is evaluated in a phase II trial using survival outcomes. In that case, the competing risk issue often arises…

Applications · Statistics 2022-03-15 Jina Park , Wenjing Hu , Ick Hoon Jin , Hao Liu , Yong Zang