A Brief Overview of Adaptive Designs for Phase I Cancer Trials.
A Brief Overview of Adaptive Designs for Phase I Cancer Trials.
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DOI：
10.3390/cancers14061566
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发表时间：
2022-03-18
期刊：
影响因子：
5.2
通讯作者：
Veledar E
中科院分区：
文献类型：
--
作者：
Saxena A;Rubens M;Ramamoorthy V;Zhang Z;Ahmed MA;McGranaghan P;Das S;Veledar E
I期癌症试验对于新药开发非常重要，可以测试通常有毒的癌症药物的安全性和最佳剂量。了解这些设计的生物统计方法对于开发对参与者安全并使用最佳剂量获得更好结果的I期研究非常重要。目前，有几个第一阶段的设计正在完善和修改，以获得更好的结果，新的设计正在不断开发。在这篇综述文章中，我们描述了几个重要的I期研究设计，以提供现有方法的简要概述。我们的评论可能有助于研究界谁的意图有一个更好的，但现有的方法的简要总结。I期研究用于估计药物的剂量-毒性特征，并为后续研究选择合适的剂量。然而，关于I期研究所用统计方法的文献非常广泛。本综述的目的是提供一个简明扼要的总结现有的和新兴的技术选择剂量，适合I期癌症试验。许多先进的统计研究提出了新的和强大的方法，自适应设计，已显示出显着的优势，比传统的剂量发现方法。越来越多的I期癌症试验使用适应性设计，特别是在研究的早期阶段。本文综述了非参数设计和基于算法的设计，如传统的3 + 3、加速滴定、基于贝叶斯算法的设计、升降设计和等渗设计。此外，我们还描述了基于参数模型的设计，如连续重新评估方法，剂量控制的升级，贝叶斯决策理论和最优设计。正在进行的研究一直专注于改进和完善现有的模型以及开发新的方法。这项研究将有助于读者吸收核心概念，并在一个旗帜下比较不同的第一阶段统计方法。然而，其他不断发展的方法需要在今后加以审查。
Phase I cancer trials are important for new drug developments to test the safety and optimal dosage of cancer drugs which are usually toxic. Understanding biostatistical methodologies of these designs is important for developing phase I studies that are both safe for the participants and which use optimal dosages for better outcomes. Currently there are several phase I designs that are being refined and modified for better outcomes and newer designs are being continuously developed. In this review article, we described several important phase I study designs to provide a brief overview of existing methods. Our review could be helpful to the research community who intent to have a better and yet a concise summary of existing methods. Phase I studies are used to estimate the dose-toxicity profile of the drugs and to select appropriate doses for successive studies. However, literature on statistical methods used for phase I studies are extensive. The objective of this review is to provide a concise summary of existing and emerging techniques for selecting dosages that are appropriate for phase I cancer trials. Many advanced statistical studies have proposed novel and robust methods for adaptive designs that have shown significant advantages over conventional dose finding methods. An increasing number of phase I cancer trials use adaptive designs, particularly during the early phases of the study. In this review, we described nonparametric and algorithm-based designs such as traditional 3 + 3, accelerated titration, Bayesian algorithm-based design, up-and-down design, and isotonic design. In addition, we also described parametric model-based designs such as continual reassessment method, escalation with overdose control, and Bayesian decision theoretic and optimal design. Ongoing studies have been continuously focusing on improving and refining the existing models as well as developing newer methods. This study would help readers to assimilate core concepts and compare different phase I statistical methods under one banner. Nevertheless, other evolving methods require future reviews.
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DOI：
10.1093/jnci/djp079
发表时间：
2009-05-20
期刊：
Journal of the National Cancer Institute
影响因子：
--
作者：
Le Tourneau C;Lee JJ;Siu LL
通讯作者：
Siu LL
DOI：
10.1177/1740774517722760
发表时间：
2017-10
期刊：
Clinical trials (London, England)
影响因子：
--
作者：
Conaway M
通讯作者：
Conaway M
DOI：
10.1007/978-4-431-55585-8_3
发表时间：
2019-01-01
期刊：
DOSE-FINDING DESIGNS FOR EARLY-PHASE CANCER CLINICAL TRIALS: A BRIEF GUIDEBOOK TO THEORY AND PRACTICE
影响因子：
--
作者：
Daimon, Takashi;Hirakawa, Akihiro;Matsui, Shigeyuki
通讯作者：
Matsui, Shigeyuki
影响因子：
1.8
作者：
Ivanova, Anastasia;Flournoy, Nancy
通讯作者：
Flournoy, Nancy
影响因子：
1.9
作者：
OQUIGLEY, J;PEPE, M;FISHER, L
通讯作者：
FISHER, L