临床研究中估计平均生存时间的参数和混合方法的比较。

IF 1.2 4区 医学 Q4 PHARMACOLOGY & PHARMACY
Yuki Nakagawa, Takashi Sozu
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引用次数: 0

摘要

平均生存时间(MST)通常用Kaplan-Meier法得到的估计生存函数曲线下的面积来估计。然而,当最大观测生存时间被截去时,由于生存函数不为零,MST无法估计。在这种情况下,使用参数和混合方法来估计MST。参数方法在整个时间内假设一个概率分布,并在一些研究中得到了评估。混合方法结合了两种方法:首先将Kaplan-Meier方法应用到指定的时间点,然后使用参数分布外推该点以外的生存曲线。对混合方法的性能评价仅限于几种数据生成机制和分析模型。本研究通过数值实验对参数化和混合化方法的性能进行了评价,并对数据生成机制和16种分析模型假设了9种概率分布。广义gamma模型和log(-log)链接函数的Royston-Parmar模型的偏差和均方根误差往往小于其他分析模型,即使在样本量较大时,分析模型的假设概率分布与数据产生机制的假设概率分布不一致。总体而言,参数方法和混合方法的性能在所有数据生成机制中都是可比较的。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Comparison of parametric and hybrid methods for estimating mean survival time in clinical study.

The mean survival time (MST) is usually estimated as the area under the curve of the estimated survival function obtained using the Kaplan-Meier method. However, when the maximum observed survival time is censored, the MST cannot be estimated because the survival function does not reach zero. In such cases, parametric and hybrid methods are used to estimate the MST. The parametric method assumes a probability distribution throughout the entire time and has been evaluated in several studies. The hybrid method combines two approaches: it first applies the Kaplan-Meier method up to a specified time point and then extrapolates the survival curve beyond this point using a parametric distribution. Evaluation of the performance of the hybrid method is limited to a few data-generating mechanisms and analysis models. This study evaluated the performance of the parametric and hybrid methods through numerical experiments, assuming nine probability distributions for the data-generating mechanism and 16 analysis models. The bias and root mean square error of the generalized gamma model and the Royston-Parmar models with the log(-log) link function tended to be smaller than those of the other analysis models, even when the assumed probability distribution of the analysis model was inconsistent with that of the data-generating mechanism when the sample size is relatively large. Overall, the performances of the parametric and hybrid methods were comparable across all the data-generating mechanisms.

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来源期刊
Journal of Biopharmaceutical Statistics
Journal of Biopharmaceutical Statistics 医学-统计学与概率论
CiteScore
2.50
自引率
18.20%
发文量
71
审稿时长
6-12 weeks
期刊介绍: The Journal of Biopharmaceutical Statistics, a rapid publication journal, discusses quality applications of statistics in biopharmaceutical research and development. Now publishing six times per year, it includes expositions of statistical methodology with immediate applicability to biopharmaceutical research in the form of full-length and short manuscripts, review articles, selected/invited conference papers, short articles, and letters to the editor. Addressing timely and provocative topics important to the biostatistical profession, the journal covers: Drug, device, and biological research and development; Drug screening and drug design; Assessment of pharmacological activity; Pharmaceutical formulation and scale-up; Preclinical safety assessment; Bioavailability, bioequivalence, and pharmacokinetics; Phase, I, II, and III clinical development including complex innovative designs; Premarket approval assessment of clinical safety; Postmarketing surveillance; Big data and artificial intelligence and applications.
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