相关论文: Explaining temporal trends in annualized relapse r…
In network meta-analysis (NMA), we synthesize all relevant evidence about health outcomes with competing treatments. The evidence may come from randomized controlled trials (RCT) or non-randomized studies (NRS) as individual participant…
An implanted device for brain-responsive neurostimulation (RNS System) is approved as an effective treatment to reduce seizures in adults with medically-refractory focal epilepsy. Clinical trials of the RNS System demonstrate…
When performing statistical analysis of single-subject fMRI data, serial correlations need to be taken into account to allow for valid inference. Otherwise, the variability in the parameter estimates might be under-estimated resulting in…
In placebo-controlled randomized trials, the post-randomization use of concomitant medications may be higher in the placebo arm than in the treatment arm. This may dilute the full benefits of the randomized drug as estimated by the…
Robins 1997 introduced marginal structural models (MSMs), a general class of counterfactual models for the joint effects of time-varying treatment regimes in complex longitudinal studies subject to time-varying confounding. In his work,…
Cancer remains a leading cause of death, highlighting the importance of effective radiotherapy (RT). Magnetic resonance-guided linear accelerators (MR-Linacs) enable imaging during RT, allowing for inter-fraction, and perhaps even…
Target trial emulation (TTE) enables causal questions to be studied with observational data when randomized controlled trials (RCTs) are infeasible. Yet treatment-effect methods often address causal estimation, missingness, and temporal…
The micro-randomized trial (MRT) is a sequential randomized experimental design to empirically evaluate the effectiveness of mobile health (mHealth) intervention components that may be delivered at hundreds or thousands of decision points.…
Covariate adjustment is desired by both practitioners and regulators of randomized clinical trials because it improves precision for estimating treatment effects. However, covariate adjustment presents a particular challenge in…
This study investigates a data-driven machine learning approach to predict membrane fouling in critically ill patients undergoing Continuous Renal Replacement Therapy (CRRT). Using time-series data from an ICU, 16 clinically selected…
In a real-life setting, little is known regarding the effectiveness of statins for primary prevention among older adults, and analysis of observational data can add crucial information on the benefits of actual patterns of use. Latent class…
Brain metastases affect approximately between 20% and 40% of cancer patients and are commonly treated with radiotherapy or radiosurgery. Early prediction of recurrence following treatment could enable timely clinical intervention and…
The primary analysis for longitudinal randomized controlled trials (RCTs) often compares treatment groups at the last timepoint, referred to as the landmark time. Assuming data are normally distributed and missing at random, the mixed model…
Magnetic Resonance Spectroscopy (MRS) is a noninvasive tool to reveal metabolic information. One challenge of 1H-MRS is the low Signal-Noise Ratio (SNR). To improve the SNR, a typical approach is to perform Signal Averaging (SA) with M…
This paper proposes a novel criterion for the allocation of patients in Phase~I dose-escalation clinical trials aiming to find the maximum tolerated dose (MTD). Conventionally, using a model-based approach the next patient is allocated to…
Responding rapidly to a patient who is demonstrating signs of imminent clinical deterioration is a basic tenet of patient care. This gave rise to a patient safety intervention philosophy known as a Rapid Response System (RRS), whereby a…
Response-Adaptive Randomization (RAR) is part of a wider class of data-dependent sampling algorithms, for which clinical trials are typically used as a motivating application. In that context, patient allocation to treatments is determined…
Cluster randomized trials (CRTs) often enroll large numbers of participants, but due to logistical and fiscal challenges, only a subset of participants may be selected for measurement of certain outcomes, and those sampled may, purposely or…
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…
Marginal structural models (MSMs) are widely used in observational studies to estimate the causal effect of time-varying treatments. Despite its popularity, limited attention has been paid to summarizing the treatment history in the outcome…