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A sequential multiple assignment randomized trial (SMART) facilitates comparison of multiple adaptive treatment strategies (ATSs) simultaneously. Previous studies have established a framework to test the homogeneity of multiple ATSs by a…

统计方法学 · 统计学 2022-11-04 Liwen Wu , Junyao Wang , Abdus S. Wahed

Objective: To exploit state variations in infant mortality, identify diagnoses that contributed to reduction of the infant mortality rate (IMR), and examine factors associated with preterm related mortality rate (PMR). Methods: Using linked…

应用统计 · 统计学 2020-12-22 Alireza Ebrahimvandi , Niyousha Hosseinichimeh , Jay Iams

In clinical or epidemiological follow-up studies, methods based on time scale indicators such as the restricted mean survival time (RMST) have been developed to some extent. Compared with traditional hazard rate indicator system methods,…

统计方法学 · 统计学 2022-05-26 Chengfeng Zhang , Hongji Wu , Baoyi Huang , Hao Yuan , Yawen Hou , Zheng Chen

Background: Multiple Sclerosis (MS), an autoimmune disease affecting millions worldwide, is characterized by its variable course, in which some patients will experience a more benign disease course and others a more active one, with the…

应用统计 · 统计学 2024-12-12 Uma Sthanu , Gary Cutter PhD

Stepped wedge cluster-randomized trial (CRTs) designs randomize clusters of individuals to intervention sequences, ensuring that every cluster eventually transitions from a control period to receive the intervention under study by the end…

统计方法学 · 统计学 2025-02-19 Alessandro Gasparini , Michael J. Crowther , Emiel O. Hoogendijk , Fan Li , Michael O. Harhay

Dose-finding studies in oncology often include an up-and-down dose transition rule that assigns a dose to each cohort of patients based on accumulating data on dose-limiting toxicity (DLT) events. In making a dose transition decision, a key…

统计方法学 · 统计学 2025-01-30 Zhiwei Zhang

Purpose: To develop and compare normal tissue complication probability (NTCP) models for recurrent brain metastases (BMs) treated with repeat single-fraction stereotactic radiosurgery (SRS), considering time-dependent discounted prior dose.…

医学物理 · 物理学 2024-09-13 Manju Sharma , Issam El Naqa , Penny K Sneed

Predicting relative risk (RR) of spatial clusters is a complex task in public health that can be achieved through various statistical and machine-learning methods for different time intervals. However, high-resolution longitudinal data is…

统计方法学 · 统计学 2025-12-23 Lyza Iamrache , Kamel Rekab , Majid Bani-Yagoub , Julia Pluta , Abdelghani Mehailia

In large observational studies, the case-cohort design is commonly used to reduce the cost associated with covariate measurement. For survival outcomes, literature has suggested that the restricted mean survival time (RMST) be a more…

统计方法学 · 统计学 2026-05-08 Andy Ni , Wei-En Lu , Bo Lu

The treatment effect in a specific subgroup is often of interest in randomized clinical trials. When the subgroup is characterized by the absence of certain post-randomization events, a naive analysis on the subset of patients without these…

应用统计 · 统计学 2018-09-12 Baldur P. Magnusson , Heinz Schmidli , Nicolas Rouyrre , Daniel O. Scharfstein

Cluster-randomized trials (CRTs) involve randomizing entire groups of participants -- called clusters -- to treatment arms but are often comprised of a limited or fixed number of available clusters. While covariate adjustment can account…

统计方法学 · 统计学 2022-11-29 Angela Y. Zhu , Nandita Mitra , Karla Hemming , Michael O. Harhay , Fan Li

This thesis presents Regenerative Rejection Sampling (RRS), a novel approximate sampling algorithm inspired by classical Rejection Sampling and Markov Chain Monte Carlo methods. The method constructs a continuous-time regenerative process…

统计计算 · 统计学 2026-04-01 Tommaso Bozzi

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

统计方法学 · 统计学 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

In stepped wedge cluster randomized trials (SW-CRTs), observations collected under the control condition are, on average, from an earlier time than observations collected under the intervention condition. In a cohort design, participants…

统计方法学 · 统计学 2023-02-23 Jale Basten , Katja Ickstadt , Nina Timmesfeld

Reinforcement learning (RL) is a type of artificial intelligence for making optimal choices. In healthcare, researchers generally use offline RL (ORL), where models are trained and evaluated from retrospective observational data. To…

机器学习 · 计算机科学 2026-04-30 Thomas Frost , Hrisheekesh Vaidya , Steve Harris

The use of amyloid-beta (A$\beta$) clearance to support regulatory approvals of drugs in Alzheimer's disease (AD) remains controversial. We evaluate A$\beta$ as a potential trial-level surrogate endpoint for clinical function in AD using a…

Background. Alzheimer's disease and related dementia (ADRD) are characterized by multiple and progressive anatomo clinical changes. Yet, modeling changes over disease course from cohort data is challenging as the usual timescales are…

统计方法学 · 统计学 2024-07-17 Jérémie Lespinasse , Carole Dufouil , Cécile Proust-Lima , the MEMENTO study group

Rheumatoid arthritis (RA) is an autoimmune condition caused when patients' immune system mistakenly targets their own tissue. Machine learning (ML) has the potential to identify patterns in patient electronic health records (EHR) to…

定量方法 · 定量生物学 2022-10-25 Shengjia Chen , Nikunj Gupta , Woodward B. Galbraith , Valay Shah , Jacopo Cirrone

Recurrent neural networks (RNNs) are commonly applied to clinical time-series data with the goal of learning patient risk stratification models. Their effectiveness is due, in part, to their use of parameter sharing over time (i.e., cells…

机器学习 · 计算机科学 2020-01-03 Jeeheh Oh , Jiaxuan Wang , Shengpu Tang , Michael Sjoding , Jenna Wiens

In confirmatory cancer clinical trials, overall survival (OS) is normally a primary endpoint in the intention-to-treat (ITT) analysis under regulatory standards. After the tumor progresses, it is common that patients allocated to the…

统计方法学 · 统计学 2022-01-19 José L. Jiménez , Julia Niewczas , Alexander Bore , Carl-Fredrik Burman