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When a novel treatment has successfully passed phase I, different options to design subsequent phase II trials are available. One approach is a single-arm trial, comparing the response rate in the intervention group against a fixed…

统计方法学 · 统计学 2020-08-03 Johannes Krisam , Dorothea Weber , Richard F. Schlenk , Meinhard Kieser

The development of statistical approaches for the joint modelling of the temporal changes of imaging, biochemical, and clinical biomarkers is of paramount importance for improving the understanding of neurodegenerative disorders, and for…

应用统计 · 统计学 2018-02-16 Marco Lorenzi , Maurizio Filippone , Daniel C. Alexander , Sebastien Ourselin

Multi-gene panel testing allows efficient detection of pathogenic variants in cancer susceptibility genes including moderate-risk genes such as ATM and PALB2. A growing number of studies examine the risk of breast cancer (BC) conferred by…

统计方法学 · 统计学 2024-02-26 Thanthirige Lakshika M. Ruberu , Danielle Braun , Giovanni Parmigiani , Swati Biswas

Assessing the causal effect of time-varying exposures on recurrent event processes is challenging in the presence of a terminating event. Our objective is to estimate both the short-term and delayed marginal causal effects of exposures on…

统计方法学 · 统计学 2025-06-10 Daniel Mork , Robert L. Strawderman , Michelle Audirac , Francesca Dominici , Ashkan Ertefaie

People are living longer than ever before, and with this arises new complications and challenges for humanity. Among the most pressing of these challenges is of understanding the role of aging in the development of dementia. This paper is…

统计方法学 · 统计学 2018-08-07 Jonathan P Williams , Curtis B Storlie , Terry M Therneau , Clifford R Jack , Jan Hannig

The Average Treatment Effect on the Treated (ATT) is a common causal parameter defined as the average effect of a binary treatment among the subset of the population receiving treatment. We propose a novel family of parameters, Generalized…

统计方法学 · 统计学 2024-10-28 Herbert Susmann , Nicholas T. Williams , Kara E. Rudolph , Iván Díaz

Randomized clinical trials are often designed to assess whether a test treatment prolongs survival relative to a control treatment. Increased patient heterogeneity, while desirable for generalizability of results, can weaken the ability of…

统计方法学 · 统计学 2020-04-30 Devan V. Mehrotra , Rachel Marceau West

Clinical decision making regarding treatments based on personal characteristics leads to effective health improvements. Machine learning (ML) has been the primary concern of diagnosis support according to comprehensive patient information.…

Critically ill patients in regular wards are vulnerable to unanticipated adverse events which require prompt transfer to the intensive care unit (ICU). To allow for accurate prognosis of deteriorating patients, we develop a novel…

机器学习 · 计算机科学 2017-05-16 Ahmed M. Alaa , Scott Hu , Mihaela van der Schaar

The dynamic environment of laboratories and clinics, with streams of data arriving on a daily basis, requires regular updates of trained machine learning models for consistent performance. Continual learning is supposed to help train models…

机器学习 · 计算机科学 2025-08-12 Zahra Ebrahimi , Raheleh Salehi , Nassir Navab , Carsten Marr , Ario Sadafi

Dynamic treatment regimes are sequential decision rules that adapt treatment according to individual time-varying characteristics and outcomes to achieve optimal effects, with applications in precision medicine, personalized…

统计方法学 · 统计学 2025-10-24 Yuanshan Gao , Yang Bai , Yifan Cui

Introduction: Modelling of relative treatment effects is an important aspect to consider when extrapolating the long-term survival outcomes of treatments. Flexible parametric models offer the ability to accurately model the observed data,…

统计方法学 · 统计学 2024-01-02 Philip Cooney , Arthur White

Dynamic prediction of causal effects under different treatment regimes conditional on an individual's characteristics and longitudinal history is an essential problem in precision medicine. This is challenging in practice because outcomes…

统计方法学 · 统计学 2023-03-07 Yizhen Xu , Jisoo Kim , Laura K. Hummers , Ami A. Shah , Scott Zeger

Medical studies for chronic disease are often interested in the relation between longitudinal risk factor profiles and individuals' later life disease outcomes. These profiles may typically be subject to intermediate structural changes due…

应用统计 · 统计学 2023-08-22 Sandra Keizer , Zhuozhao Zhan , Vasan S. Ramachandran , Edwin R. van den Heuvel

Approving and assessing new drugs is complex because multiple criteria must be considered simultaneously. A common approach is benefit-risk analysis, often conducted within a Bayesian framework to account for uncertainty and combine data…

统计方法学 · 统计学 2026-05-05 Konstantinos Vamvourellis , Konstantinos Kalogeropoulos , Lawrence Phillips

Emerging AI systems in behavioral health and psychiatry use multi-step or multi-agent LLM pipelines for tasks like assessing self-harm risk and screening for depression. However, common evaluation approaches, like LLM-as-a-judge, do not…

机器学习 · 计算机科学 2026-04-27 Meghana Karnam , Ananya Joshi

Bone Marrow Transplant, a gradational rescue for a wide range of disorders emanating from the bone marrow, is an efficacious surgical treatment. Several risk factors, such as post-transplant illnesses, new malignancies, and even organ…

The aim of the systematic review was to assess recently published studies on diagnostic test accuracy of glioblastoma treatment response monitoring biomarkers in adults, developed through machine learning (ML). Articles were searched for…

Precision medicine leverages patient heterogeneity to estimate individualized treatment regimens, formalized, data-driven approaches designed to match patients with optimal treatments. In the presence of competing events, where multiple…

统计方法学 · 统计学 2024-11-14 Christina W. Zhou , Nikki L. B. Freeman , Katharine L. McGinigle , Michael R. Kosorok

Estimating heterogeneous treatment effects in survival settings is complicated by right censoring as well as the time-varying nature of the estimand. While the conditional average treatment effect (CATE) provides a natural target, most…

统计方法学 · 统计学 2026-04-14 Yuming Sun , Jian Kang , Yi Li