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We argue that randomized controlled trials (RCTs) are special even among settings where average treatment effects are identified by a nonparametric unconfoundedness assumption. This claim follows from two results of Robins and Ritov (1997):…

Methodology · Statistics 2021-09-28 P. M. Aronow , James M. Robins , Theo Saarinen , Fredrik Sävje , Jasjeet Sekhon

Non-adherence to assigned treatment is a common issue in cluster randomised trials (CRTs). In these settings, the efficacy estimand may be also of interest. Many methodological contributions in recent years have advocated using instrumental…

Methodology · Statistics 2018-12-05 Schadrac C. Agbla , Bianca De Stavola , Karla DiazOrdaz

Background: Reliable prediction of clinical progression over time can improve the outcomes of depression. Little work has been done integrating various risk factors for depression, to determine the combinations of factors with the greatest…

Machine Learning · Statistics 2023-07-06 Runa Bhaumik , Jonathan Stange

Age is one of the major known risk factors for Alzheimer's Disease (AD). Detecting AD early is crucial for effective treatment and preventing irreversible brain damage. Brain age, a measure derived from brain imaging reflecting structural…

Image and Video Processing · Electrical Eng. & Systems 2024-05-07 Jay Shah , Md Mahfuzur Rahman Siddiquee , Yi Su , Teresa Wu , Baoxin Li

Propensity Score Matching (PSM) is an useful method to reduce the impact ofTreatment - Selection Bias in the estimation of causal effects in observational studies. After matching, the PSM significantly reduces the sample under…

Methodology · Statistics 2019-02-01 Daniel García Iglesias

Major depressive disorder (MDD) is a heterogeneous condition; multiple underlying neurobiological substrates could be associated with treatment response variability. Understanding the sources of this variability and predicting outcomes has…

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…

Restricted mean survival time (RMST) is gaining attention as a measure to quantify the treatment effect on survival outcomes in randomized clinical trials. Several methods to determine sample size based on the RMST-based tests have been…

Methodology · Statistics 2022-12-19 Satoshi Hattori , Hajime Uno

Risk prediction is central to both clinical medicine and public health. While many machine learning models have been developed to predict mortality, they are rarely applied in the clinical literature, where classification tasks typically…

Machine Learning · Statistics 2017-12-05 Maggie Makar , Marzyeh Ghassemi , David Cutler , Ziad Obermeyer

Subgroup analyses within randomized controlled trials are often underpowered due to limited sample sizes. We address this challenge by leveraging trial participants outside the subgroup of interest to augment estimation within the subgroup.…

Methodology · Statistics 2026-05-18 Sky Qiu , Nerissa Nance , Rachael Phillips , Jens Tarp , Maya Petersen , Mark van der Laan

Recently, Mackenzie et al. reported incidence and prevalence of multiple sclerosis (MS) in the UK from 1990-2010. Authors of this report indicated that they could not explain the cause of decreasing MS incidence trend in the UK during this…

Other Quantitative Biology · Quantitative Biology 2013-12-18 Seyed Aidin Sajedi , Fahimeh Abdollahi

With the uptake of targeted therapies, instead of the "one-fits-all" approach, modern randomized clinical trials (RCTs) often aim to develop treatments that target a subgroup of patients. Motivated by analyzing the Age-Related Eye Disease…

Applications · Statistics 2020-03-25 Yue Wei , Jason C. Hsu , Wei Chen , Emily Y. Chew , Ying Ding

Background: Incidence of adverse outcome events rises as patients with advanced illness approach end-of-life. Exposures that tend to occur near end-of-life, e.g., use of wheelchair, oxygen therapy and palliative care, may therefore be found…

The difference in restricted mean survival time (RMST) is a clinically meaningful measure to quantify treatment effect in randomized controlled trials, especially when the proportional hazards assumption does not hold. Several frequentist…

Accurate prediction of future risk and disease progression in sepsis is clinically important for early warning and timely intervention in intensive care. However, short-window sepsis prediction remains challenging, because shorter…

Machine Learning · Computer Science 2026-04-17 Honglin Guo , Rihao Chang , He Jiao , Weizhi Nie , Zhongheng Zhang , Yuehao Shen

Brain Metastases (BM) are a large contributor to mortality of patients with cancer. They are treated with Stereotactic Radiosurgery (SRS) and monitored with Magnetic Resonance Imaging (MRI) at regular follow-up intervals according to…

Computer Vision and Pattern Recognition · Computer Science 2025-09-09 Lorenz Achim Kuhn , Daniel Abler , Jonas Richiardi , Andreas F. Hottinger , Luis Schiappacasse , Vincent Dunet , Adrien Depeursinge , Vincent Andrearczyk

Introduction Quantum Convolutional Neural Network (QCNN)-Long Short-Term Memory (LSTM) models were studied to provide sequential relationships for each timepoint in MRIs of patients with Multiple Sclerosis (MS). In this pilot study, we…

Machine Learning · Computer Science 2024-01-23 John D. Mayfield , Issam El Naqa

Response-adaptive randomization (RAR) has been studied extensively in conventional, single-stage clinical trials, where it has been shown to yield ethical and statistical benefits, especially in trials with many treatment arms. However, RAR…

Methodology · Statistics 2024-01-09 Peter Norwood , Marie Davidian , Eric Laber

Response-adaptive randomisation (RAR) can considerably improve the chances of a successful treatment outcome for patients in a clinical trial by skewing the allocation probability towards better performing treatments as data accumulates.…

Applications · Statistics 2017-03-14 Sofia S. Villar , Jack Bowden , James Wason

Artificial intelligence (AI) has increasingly transformed medical prognostics by enabling rapid and accurate analysis across imaging and pathology. However, the investigation of machine learning predictions applied to prospectively…