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相关论文: On Assessing Overall Survival (OS) in Oncology Stu…

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Measuring treatment efficacy in mixture of subgroups from a randomized clinical trial is a fundamental problem in personalized medicine development, in deciding whether to treat the entire patient population or to target a subgroup. We show…

统计方法学 · 统计学 2014-09-03 Ying Ding , Hui-Min Lin , Jason C. Hsu

Advances in anticancer therapies have significantly contributed to declining death rates in certain disease and clinical settings. However, they have also made it difficult to power a clinical trial in these settings with overall survival…

统计方法学 · 统计学 2025-12-09 Godwin Yung , Kaspar Rufibach , Marcel Wolbers , Mark Yan , Jue Wang

The development of oncology drugs progresses through multiple phases, where after each phase a decision is made about whether to move a molecule forward. Early phase efficacy decisions are often made on the basis of single arm studies based…

应用统计 · 统计学 2023-04-17 Ulrich Beyer , David Dejardin , Matthias Meller , Kaspar Rufibach , Hans Ulrich Burger

In oncology clinical trials, characterizing the long-term overall survival (OS) benefit for an experimental drug or treatment regimen (experimental group) is often unobservable if some patients in the control group switch to drugs in the…

应用统计 · 统计学 2019-08-05 Yuqing Xu , Meijing Wu , Weili He , Qiming Liao , Yabing Mai

In oncology, conduct well-powered time-to-event randomized clinical trials may be challenging due to limited patietns number. Many designs for single-arm trials (SATs) have recently emerged as an alternative to overcome this issue. They…

应用统计 · 统计学 2026-04-10 Chloé Szurewsky , Guosheng Yin , Gwénaël Le Teuff

Proportional hazards are a common assumption when designing confirmatory clinical trials in oncology. With the emergence of immunotherapy and novel targeted therapies, departure from the proportional hazard assumption is not rare in…

统计方法学 · 统计学 2020-08-27 José L. Jiménez

Overall survival (OS) time prediction is one of the most common estimates of the prognosis of gliomas and is used to design an appropriate treatment planning. State-of-the-art (SOTA) methods for OS time prediction follow a pre-hoc approach…

计算机视觉与模式识别 · 计算机科学 2021-02-23 Renato Hermoza , Gabriel Maicas , Jacinto C. Nascimento , Gustavo Carneiro

Restricted mean survival time (RMST) offers a compelling nonparametric alternative to hazard ratios for right-censored time-to-event data, particularly when the proportional hazards assumption is violated. By capturing the total event-free…

统计方法学 · 统计学 2025-01-28 Jinghao Sun , Douglas E. Schaubel , Eric J. Tchetgen Tchetgen

Overall survival (OS) is the gold standard for assessing patient benefit and cost-effectiveness of new cancer drugs. However, it is often difficult to use OS as the primary endpoint in randomized clinical trials (RCTs) for patients with…

统计方法学 · 统计学 2026-04-23 Sidi Wang , Kelley Kidwell , Bo Huang , Satrajit Roychoudhury

We conducted a systematic comparison of statistical methods used for the analysis of time-to-event outcomes under various proportional and nonproportional hazard (NPH) scenarios. Our study used data from recently published oncology trials…

应用统计 · 统计学 2025-02-12 Xinyu Zhang , Erich J. Greene , Ondrej Blaha , Wei Wei

Predicting cancer treatment outcomes requires models that are both accurate and interpretable, particularly in the presence of heterogeneous clinical data. While large language models (LLMs) have shown strong performance in biomedical NLP,…

计算与语言 · 计算机科学 2025-10-21 Raghu Vamshi Hemadri , Geetha Krishna Guruju , Kristi Topollai , Anna Ewa Choromanska

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

Background: Often when undertaking meta-analyses of time-to-event (TTE) outcomes, especially in a Health Technology Assessment context, a hazard ratio (HR) scale is used. However, issues arise when there is evidence of non-proportional…

统计方法学 · 统计学 2026-05-21 Rhiannon K Owen , Keith R Abrams

An accurately identified maximum tolerated dose (MTD) serves as the cornerstone of successful subsequent phases in oncology drug development. Bayesian logistic regression model (BLRM) is a popular and versatile model-based dose-finding…

统计方法学 · 统计学 2021-05-17 Hongtao Zhang , Alan Y Chiang , Jixian Wang

When planning an oncology clinical trial, the usual approach is to assume proportional hazards and even an exponential distribution for time-to-event endpoints. Often, besides the gold-standard endpoint overall survival (OS),…

应用统计 · 统计学 2023-12-19 Alexandra Erdmann , Jan Beyersmann , Kaspar Rufibach

Duration of response (DOR) and time to response (TTR) are typically evaluated as secondary endpoints in early-stage clinical studies in oncology when efficacy is assessed by the best overall response (BOR) and presented as the overall…

Hazard ratios are ubiquitously used in time to event analysis to quantify treatment effects. Although hazard ratios are invaluable for hypothesis testing, other measures of association, both relative and absolute, may be used to fully…

统计方法学 · 统计学 2020-11-02 Federico Ambrogi , Simona Iacobelli , Per Kragh Andersen

The primary endpoint in oncology is usually overall survival, where differences between therapies may only be observable after many years. To avoid withholding of a promising therapy, preliminary approval based on a surrogate endpoint is…

统计方法学 · 统计学 2022-05-25 Samuel Kilian , Johannes Krisam , Meinhard Kieser

Five-year cancer survival rates are widely reported and often interpreted to mean that early detection saves lives, that a late fatal diagnosis would have been prevented by earlier detection, and that increasing survival over time proves…

统计方法学 · 统计学 2026-03-23 Allen B. Downey
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