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As a future trend of healthcare, personalized medicine tailors medical treatments to individual patients. It requires to identify a subset of patients with the best response to treatment. The subset can be defined by a biomarker (e.g.…

Methodology · Statistics 2021-08-16 Yitao Lu , Julie Zhou , Li Xing , Xuekui Zhang

The concept of personalised medicine in cancer therapy is becoming increasingly important. There already exist drugs administered specifically for patients with tumours presenting well-defined mutations. However, the field is still in its…

Biomolecules · Quantitative Biology 2024-08-26 Abbi Abdel-Rehim , Oghenejokpeme Orhobor , Gareth Griffiths , Larisa Soldatova , Ross D. King

Delineating and planning with respect to regions suspected to contain microscopic tumor cells is an inherently uncertain task in radiotherapy. The recently proposed \textit{clinical target distribution} (CTD) is an alternative to the…

Medical Physics · Physics 2023-04-26 Ivar Bengtsson , Anders Forsgren , Albin Fredriksson

Clinical trials constitute a critical yet exceptionally challenging and costly stage of drug development (\$2.6B per drug), where protocols are encoded as complex natural language documents, motivating the use of AI systems beyond manual…

Artificial Intelligence · Computer Science 2026-04-06 Sixue Xing , Kerui Wu , Xuanye Xia , Meng Jiang , Jintai Chen , Tianfan Fu

Oncology dose-finding trials are shifting from identifying the maximum tolerated dose (MTD) to determining the optimal biological dose (OBD), driven by the need for efficient methods that consider both toxicity and efficacy. This is…

Methodology · Statistics 2025-04-01 Hao Sun , Jieqi Tu , Revathi Ananthakrishnan , Eunhee Kim

The FDA's Project Optimus initiative emphasizes patient-centered dose selection in oncology that balances efficacy and safety. We develop a framework for randomized dose optimization studies that uses clinically interpretable utility scores…

Applications · Statistics 2026-03-24 Xuemin Gu , Cong Xu , Lei Xu , Ying Yu

To accurately verify the dose of intensity-modulated radiation therapy (IMRT), we have used a global optimization method to investigate a new dose-verification algorithm. In practical application of this quality assurance (QA) procedure,…

Medical Physics · Physics 2007-05-23 Dong Hyun Park , Sung-Yong Park , Dahl Park , Tae-Hyun Kim , Kyung Hwan Shin , Dae Yong Kim , Kwan-Ho Cho

Optimal design of a Phase I cancer trial can be formulated as a stochastic optimization problem. By making use of recent advances in approximate dynamic programming to tackle the problem, we develop an approximation of the Bayesian optimal…

Methodology · Statistics 2010-12-01 Jay Bartroff , Tze Leung Lai

Radiotherapy is a primary treatment for cancers with the aim of applying sufficient radiation dose to the planning target volume (PTV) while minimizing dose hazards to the organs at risk (OARs). Convolutional neural networks (CNNs) have…

Image and Video Processing · Electrical Eng. & Systems 2024-02-08 Lu Wen , Qihun Zhang , Zhenghao Feng , Yuanyuan Xu , Xiao Chen , Jiliu Zhou , Yan Wang

Combination of several anti-cancer treatments has typically been presumed to have enhanced drug activity. Motivated by a real clinical trial, this paper considers phase I-II dose finding designs for dual-agent combinations, where one main…

Methodology · Statistics 2023-05-09 José L. Jiménez , Haiyan Zheng

Purpose: The early identification of maximum tolerated dose (MTD) in phase I trial leads to faster progression to a phase II trial or an expansion cohort to confirm efficacy. Methods: We propose a novel adaptive design for identifying MTD…

Methodology · Statistics 2021-10-07 Masahiro Kojima

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…

Methodology · Statistics 2024-04-15 Runjia Li , Liwen Wu , Rachael Liu , Jianchang Lin

Leveraging preclinical animal data for a phase I first-in-man trial is appealing yet challenging. A prior based on animal data may place large probability mass on values of the dose-toxicity model parameter(s), which appear infeasible in…

Applications · Statistics 2020-04-15 Haiyan Zheng , Lisa V. Hampson

One common approach for dose optimization is a two-stage design, which initially conducts dose escalation to identify the maximum tolerated dose (MTD), followed by a randomization stage where patients are assigned to two or more doses to…

Methodology · Statistics 2024-11-11 Yixuan Zhao , Rachael Liu , Jianchang Lin , Ying Yuan

Patients who are seriously ill may ask doctors to treat them with unapproved medication, about which not much is known, or else with known medication in a high dosage. Apart from strict legal constraints such cases may involve difficult…

Other Statistics · Statistics 2018-04-13 F. Thomas Bruss

An unprecedented number of new cancer targets are in development, and most are being developed in combination therapies. Early oncology development is strategically challenged in choosing the best combinations to move forward to late stage…

Applications · Statistics 2021-01-08 Linda Z. Sun , Cai , Wu , Xiaoyun , Li , Cong Chen , Emmett V. Schmidt

Large variability between cell lines brings a difficult optimization problem of drug selection for cancer therapy. Standard approaches use prediction of value for this purpose, corresponding e.g. to expected value of their distribution.…

Quantitative Methods · Quantitative Biology 2022-09-15 Jarek Duda

When the early stopping parameter n.earlystop is relatively small or the cohortsize value is not optimized via simulation, it may be better to use p.tox < 1.4 * target.DLT.rate, or try out different cohort sizes, or increase n.earlystop,…

Medical Physics · Physics 2024-03-18 Rong Lu

Aims: Combinations of treatments can offer additional benefit over the treatments individually. However, trials of these combinations are lower priority than the development of novel therapies, which can restrict funding, timelines and…

A general framework is proposed for Bayesian model-based designs of Phase I cancer trials, in which a general criterion for coherence (Cheung, 2005) of a design is also developed. This framework can incorporate both "individual" and…

Methodology · Statistics 2011-08-08 Jay Bartroff , Tze Leung Lai