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Dual agent dose-finding trials study the effect of a combination of more than one agent, where the objective is to find the Maximum Tolerated Dose Combination (MTC), the combination of doses of the two agents that is associated with a…

应用统计 · 统计学 2025-02-10 Helen Barnett , Oliver Boix , Dimitris Kontos , Thomas Jaki

The primary goal of a two-stage Phase I/II trial is to identify the optimal dose for the following large-scale Phase III trial. Recently, Phase I dose-finding designs have shifted from identifying the maximum tolerated dose (MTD) to the…

统计方法学 · 统计学 2025-01-16 Hao Sun , Jerry Li

The use of `backfilling', assigning additional patients to doses deemed safe, in phase I dose-escalation studies has been used in practice to collect additional information on the safety profile, pharmacokinetics and activity of a drug.…

应用统计 · 统计学 2022-04-22 Helen Barnett , Oliver Boix , Dimitris Kontos , Thomas Jaki

When selecting from a list of potential candidates, it is important to ensure not only that the selected items are of high quality, but also that they are sufficiently dissimilar so as to both avoid redundancy and to capture a broader range…

统计方法学 · 统计学 2026-02-25 Yash Nair , Ying Jin , James Yang , Emmanuel Candes

In many clinical trials, individuals in different subgroups have experience differential treatment effects. This leads to individualized differences in treatment benefit. In this article, we introduce the general concept of predictive…

统计方法学 · 统计学 2018-07-11 Debashis Ghosh , Youngjoo Cho

Cancer is a number of related yet highly heterogeneous diseases. Correct identification of cancer subtypes is critical for clinical decisions. The advance in sequencing technologies has made it possible to study cancer based on abundant…

应用统计 · 统计学 2018-11-27 Xiaochun Chen , Honggang Wang , Donghui Yan

Next-generation sequencing technologies now constitute a method of choice to measure gene expression. Data to analyze are read counts, commonly modeled using Negative Binomial distributions. A relevant issue associated with this…

统计方法学 · 统计学 2014-11-10 Elisabetta Bonafede , Franck Picard , Stéphane Robin , Cinzia Viroli

One primary goal of precision medicine is to estimate the individualized treatment rules (ITRs) that optimize patients' health outcomes based on individual characteristics. Health studies with multiple treatments are commonly seen in…

统计方法学 · 统计学 2025-05-07 Xuqiao Li , Qiuyan Zhou , Ying Wu , Ying Yan

The assessment of imaging biomarkers is critical for advancing precision medicine and improving disease characterization. Despite the availability of methods to derive disease heterogeneity metrics in imaging studies, a robust framework for…

2-in-1 design (Chen et al. 2018) is becoming popular in oncology drug development, with the flexibility of using different endpoints at different decision time. Based on the observed interim data, sponsors choose either to seamlessly…

统计方法学 · 统计学 2024-04-15 Runjia Li , Liwen Wu , Rachael Liu , Jianchang Lin

Dose-finding clinical trials in oncology aim to estimate the maximum tolerated dose (MTD), based on safety traditionally obtained from the clinician's perspective. While the collection of patient-reported outcomes (PROs) has been advocated…

应用统计 · 统计学 2023-04-03 Anaïs Andrillon , Lucie Biard , Shing M. Lee

Given the prominence of targeted therapy and immunotherapy in cancer treatment, it becomes imperative to consider heterogeneity in patients' responses to treatments, which contributes greatly to the widely used proportional hazard…

统计方法学 · 统计学 2024-09-01 Powei Chen , Siying Hu , Haojin Zhou

Determining the optimal initial dose for warfarin is a critically important task. Several factors have an impact on the therapeutic dose for individual patients, such as patients' physical attributes (Age, Height, etc.), medication profile,…

机器学习 · 计算机科学 2019-03-25 Ashkan Sharabiani , Adam Bress , William Galanter , Rezvan Nazempour , Houshang Darabi

In the genomic era, the identification of gene signatures associated with disease is of significant interest. Such signatures are often used to predict clinical outcomes in new patients and aid clinical decision-making. However, recent…

统计方法学 · 统计学 2019-03-27 Naim U. Rashid , Quefeng Li , Jen Jen Yeh , Joseph G. Ibrahim

The effectiveness of personalized oncology treatments ultimately depends on whether outcomes can be causally attributed to the treatment. Advances in precision oncology have improved molecular profiling of individuals, and tailored…

应用统计 · 统计学 2025-09-29 Lydia Jang , Stefan Konigorski

The long-term efficacy of targeted therapeutics for cancer treatment can be significantly limited by the type of therapy and development of drug resistance, inter alia. Experimental studies indicate that the factors enhancing acquisition of…

组织与器官 · 定量生物学 2021-02-16 Joseph Malinzi , Kevin Bosire Basita , Sara Padidar , Henry A. Adeola

Risk stratification is a key tool in clinical decision-making, yet current approaches often fail to translate sophisticated survival analysis into actionable clinical criteria. We present a novel method for unsupervised machine learning…

Gene expression analysis is a critical method for cancer classification, enabling precise diagnoses through the identification of unique molecular signatures associated with various tumors. Identifying cancer-specific genes from gene…

Matching cancer patients to clinical trials is essential for advancing treatment and patient care. However, the inconsistent format of medical free text documents and complex trial eligibility criteria make this process extremely…

Multi-Cancer Early Detection (MCED) testing with tissue localization aims to detect and identify multiple cancer types from a single blood sample. Such tests have the potential to aid clinical decisions and significantly improve health…

统计方法学 · 统计学 2025-05-28 A. Gregory DiRienzo , Elie Massaad , Hutan Ashrafian