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Osteosarcoma is the most common primary bone cancer whose standard treatment includes pre-operative chemotherapy followed by resection. Chemotherapy response is used for predicting prognosis and further management of patients. Necrosis is…

Cancer patients may undergo lengthy and painful chemotherapy treatments, comprising several successive regimens or plans. Treatment inefficacy and other adverse events can lead to discontinuation (or failure) of these plans, or prematurely…

定量方法 · 定量生物学 2025-12-15 Muhammad Usamah Shahid , Muddassar Farooq

Optimal treatment regimes (OTR) are individualised treatment assignment strategies that identify a medical treatment as optimal given all background information available on the individual. We discuss Bayes optimal treatment regimes…

统计方法学 · 统计学 2018-10-02 Thomas Klausch , Peter van de Ven , Tim van de Brug , Mark A. van de Wiel , Johannes Berkhof

Many phase II clinical trials have used survival outcomes as the primary endpoints in recent decades. Suppose the radiotherapy is evaluated in a phase II trial using survival outcomes. In that case, the competing risk issue often arises…

应用统计 · 统计学 2022-03-15 Jina Park , Wenjing Hu , Ick Hoon Jin , Hao Liu , Yong Zang

Although the survival rate of cancer patients has significantly increased due to advances in anti-cancer therapeutics, one of the major side effects of these therapies, particularly radiotherapy, is the potential manifestation of…

定量方法 · 定量生物学 2014-11-07 Kamran Kaveh , Venkata S. K. Manem , Mohammad Kohandel , Siv Sivaloganathan

In clinical trials, patients may discontinue treatments prematurely, breaking the initial randomization and, thus, challenging inference. Stakeholders in drug development are generally interested in going beyond the Intention-To-Treat (ITT)…

Estimating causal effects for survival outcomes in the high-dimensional setting is an extremely important topic for many biomedical applications as well as areas of social sciences. We propose a new orthogonal score method for treatment…

统计方法学 · 统计学 2024-12-04 Jue Hou , Jelena Bradic , Ronghui Xu

Treatment switching in a randomized controlled trial is said to occur when a patient randomized to one treatment arm switches to another treatment arm during follow-up. This can occur at the point of disease progression, whereby patients in…

统计方法学 · 统计学 2021-03-24 Andrew Ying , Eric J. Tchetgen Tchetgen

We conducted a proof-of-concept evaluation of individualized treatment effect (ITE) estimation using survival data from a randomized trial of 475 men with advanced prostate cancer treated with high- versus low-dose diethylstilbestrol (DES).…

应用统计 · 统计学 2025-08-26 J. T. Korley

Due to the presence of multiple types of adverse events with different levels of severity, the analysis of longitudinal toxicity data is a difficult task in cancer studies. In this work, a novel approach based on latent Markov models and…

应用统计 · 统计学 2024-12-05 Marta Spreafico , Francesca Ieva , Marta Fiocco

Chemotherapy is one of the most important therapeutic options used to treat human cancers, either alone or in combination with radiation therapy and surgery. Recent studies have indicated that intra-tumoural heterogeneity has a significant…

组织与器官 · 定量生物学 2014-07-04 Gibin G Powathil , Mark AJ Chaplain , Maciej Swat

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…

统计方法学 · 统计学 2025-06-11 Ahmed Boughdiri , Julie Josse , Erwan Scornet

In biomedical studies, estimating drug effects on chronic diseases requires a long follow-up period, which is difficult to meet in randomized clinical trials (RCTs). The use of a short-term surrogate to replace the long-term outcome for…

统计方法学 · 统计学 2023-06-27 Wenjie Hu , Xiaohua Zhou , Peng Wu

In the presence of heterogeneity between the randomized controlled trial (RCT) participants and the target population, evaluating the treatment effect solely based on the RCT often leads to biased quantification of the real-world treatment…

统计方法学 · 统计学 2022-10-05 Dasom Lee , Shu Yang , Xiaofei Wang

One of the most common ways researchers compare survival outcomes across treatments when confounding is present is using Cox regression. This model is limited by its underlying assumption of proportional hazards; in some cases, substantial…

应用统计 · 统计学 2021-02-02 Elizabeth A. Handorf , Marc Smaldone , Sujana Movva , Nandita Mitra

Chemotherapy dose optimization can be formulated as a dynamic treatment regime, requiring sequential decisions under uncertainty that must balance tumor suppression against toxicity. However, most reinforcement learning approaches assume…

机器学习 · 计算机科学 2026-05-05 Firas Mohamed Elamine Kiram , Imane Youkana , Rachida Saouli , Gian Antonio Susto , Laid Kahloul

In this work, we have investigated the sensitivity of the effectiveness (TCP) of molecular radiotherapy (MRT) treatment to uncertainties of the dose rate curves that may appear when reconstructing those curves. We generated different dose…

医学物理 · 物理学 2025-02-03 Pedro Otero-Casal , Aldán Baliño , Sara Neira , Faustino Gómez , Juan Pardo-Montero

A crucial task for a randomized controlled trial (RCT) is to specify a statistical method that can yield an efficient estimator and powerful test for the treatment effect. A novel and effective strategy to obtain efficient and powerful…

Individualized treatment regimes (ITRs) aim to improve clinical outcomes by assigning treatment based on patient-specific characteristics. However, existing methods often struggle with high-dimensional covariates, limiting accuracy,…

机器学习 · 统计学 2026-01-13 Sungtaek Son , Eardi Lila , Kwun Chuen Gary Chan

Radiotherapy is a commonly used treatment for cancer and is usually given in varying doses. At low radiation doses relatively few cells die as a direct response to radiation but secondary radiation effects such as DNA mutation or bystander…

定量方法 · 定量生物学 2016-06-16 Gibin G Powathil , Alastair J Munro , Mark AJ Chaplain , Maciej Swat
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